User login
FDA to step up oversight of cosmetics, assess ‘forever chemicals’
They are also preparing to assess potential risks of so-called forever chemicals in these products.
The Food and Drug Administration last year gained new authority over cosmetics when Congress passed the Modernization of Cosmetics Regulation Act of 2022 (MoCRA) by adding this bill to a December budget package.
“On average, consumers in the U.S. use six to 12 cosmetics products daily. But, until recently the FDA didn’t have the authority to require manufacturers to submit cosmetic product listings, including a list of ingredients used in these products, or register the facilities where they were produced,” Namandjé Bumpus, PhD, FDA’s chief scientist, said in a press release.
In the statement, the FDA announced the release of a draft guidance document that is intended to help companies comply with the transparency requirements slated to kick in this December. The agency is accepting comments on this draft guidance through Sept. 7.
“Later this year, registration and listing of cosmetic product facilities and products will become a requirement, making information about cosmetic products, including the ingredients used in products and the facilities where they are produced, readily available to the agency,” Dr. Bumpus said.
The products, according to the FDA statement, include makeup, nail polishes, shaving creams, other grooming products, perfumes, face and body cleansers, hair products, moisturizers, and other skin care items.
MoCRA “represents a sea change in how FDA regulates the cosmetics industry,” attorneys Frederick R. Ball, Alyson Walker Lotman, and Kelly A. Bonner, wrote in an article for the Food and Drug Law Institute published in spring 2023.
The FDA has called the MoCRA law “the most significant expansion” of its authority to regulate cosmetics since the Federal Food, Drug, and Cosmetic Act was passed in 1938.
The agency is in the process of expanding its staff to carry out newly authorized duties, including the tracking of adverse events. The FDA budget request for fiscal 2024, which begins Oct. 1, seeks $5 million for work needed to implement MoCRA.
PFAS, or ‘forever chemicals’
Some of the requested FDA funding is intended to prepare the agency to assess the use of per-and polyfluoroalkyl substances (PFAS) in cosmetics.
MoCRA sets a 3-year deadline for the FDA to issue an assessment of the use and potential risks of PFAS in cosmetics products. PFAS are sometimes added as ingredients in some cosmetic products, including lotions, cleansers, nail polish, shaving cream, foundation, lipstick, eyeliner, eyeshadow, and mascara, according to the FDA. Sometimes the presence of PFAS in cosmetics is unintentional and is the result of impurities in raw materials or is due to the breakdown of ingredients, the FDA said.
The FDA’s website says that so far, the available research doesn’t allow for “definitive conclusions about the potential health risks of PFAS in cosmetics.”
The Centers for Disease Control and Prevention has stated that research has suggested potential links between high levels of certain PFAS, in general, with increased cholesterol levels, changes in liver enzyme levels, increased risk of hypertension or preeclampsia in pregnant women, and increased risk of kidney or testicular cancer.
PFAS compounds often are used to resist grease, oil, water, and heat in industrial settings. They are used in thousands of products, from nonstick cookware to firefighting foams and protective gear, because they can reduce friction, according to a National Academies of Sciences, Engineering, and Medicine report on PFAS that was issued last year.
PFAS are known as “forever chemicals” because they contain a carbon-fluorine bond, which does not break naturally. Even when PFAS are transformed in the body, they can assume other forms of PFAS that preserve the troublesome carbon-fluorine bond. With PFAS, the human body is confronted with a substance it doesn’t have the tools to process.
This is in contrast to proteins and carbohydrates, which are in a sense prepackaged for relatively easy disassembly in the human body. Many of these compounds have weak links that enzymes and stomach acid can take apart, such as sulfur-to-sulfur (disulfide) bonds. That’s why protein-based biotech drugs are injected instead of administered as pills. The ultimate goal of this digestion is for the body to gain energy from these compounds.
But with PFAS, the body faces the challenge of carbon-fluorine bonds that are very hard to break down, and there is no payoff for these efforts, Graham F. Peaslee, PhD, professor of physics at the University of Notre Dame (Indiana), told this news organization.
“Nothing will naturally eat it because when you break the bond, it’s like eating celery,” he said. “You use more calories to eat the celery than you gain back from it.”
Interest from a U.S. senator
Dr. Peaslee was one of the authors of a 2021 article about PFAS in cosmetics that appeared in the journal Environmental Science and Technology Letters.
In the article, Dr. Peaslee and colleagues reported on their screening of 231 cosmetic products purchased in the United States and Canada using particle-induced gamma-ray emission spectroscopy. They found cases of undisclosed PFAS in cosmetic products. Foundations, mascaras, and lip products were noted as being especially problematic.
Sen. Susan Collins (R-ME) cited Dr. Peaslee’s article in a 2021 floor speech as she argued for having the FDA ban the intentional addition of PFAS to cosmetics.
“The findings of this study are particularly alarming, as many of these products are subject to direct human exposure,” Sen. Collins said. “For example, lipstick is often inadvertently ingested, and mascara is sometimes absorbed through tear ducts.”
In addition, workers at cosmetics plants may be exposed to PFAS and discarded cosmetics that have these compounds, which could potentially contaminate drinking water, Sen. Collins said. In 2021, she introduced legislation seeking a ban on PFAS that are intentionally added to cosmetics. That legislation did not advance through the Senate.
But the Senate Appropriations Committee, on which Sen. Collins is the ranking Republican, wants the FDA to keep a ban on PFAS in mind.
The Senate Agriculture Appropriations subcommittee, which oversees the FDA’s budget, raised the issue of PFAS and cosmetics in a June report. The FDA should develop a plan outlining research needed to inform “regulatory decision making, including potential development of a proposed rule to ban intentionally added PFAS substances in cosmetics,” the subcommittee said.
A version of this article first appeared on Medscape.com.
They are also preparing to assess potential risks of so-called forever chemicals in these products.
The Food and Drug Administration last year gained new authority over cosmetics when Congress passed the Modernization of Cosmetics Regulation Act of 2022 (MoCRA) by adding this bill to a December budget package.
“On average, consumers in the U.S. use six to 12 cosmetics products daily. But, until recently the FDA didn’t have the authority to require manufacturers to submit cosmetic product listings, including a list of ingredients used in these products, or register the facilities where they were produced,” Namandjé Bumpus, PhD, FDA’s chief scientist, said in a press release.
In the statement, the FDA announced the release of a draft guidance document that is intended to help companies comply with the transparency requirements slated to kick in this December. The agency is accepting comments on this draft guidance through Sept. 7.
“Later this year, registration and listing of cosmetic product facilities and products will become a requirement, making information about cosmetic products, including the ingredients used in products and the facilities where they are produced, readily available to the agency,” Dr. Bumpus said.
The products, according to the FDA statement, include makeup, nail polishes, shaving creams, other grooming products, perfumes, face and body cleansers, hair products, moisturizers, and other skin care items.
MoCRA “represents a sea change in how FDA regulates the cosmetics industry,” attorneys Frederick R. Ball, Alyson Walker Lotman, and Kelly A. Bonner, wrote in an article for the Food and Drug Law Institute published in spring 2023.
The FDA has called the MoCRA law “the most significant expansion” of its authority to regulate cosmetics since the Federal Food, Drug, and Cosmetic Act was passed in 1938.
The agency is in the process of expanding its staff to carry out newly authorized duties, including the tracking of adverse events. The FDA budget request for fiscal 2024, which begins Oct. 1, seeks $5 million for work needed to implement MoCRA.
PFAS, or ‘forever chemicals’
Some of the requested FDA funding is intended to prepare the agency to assess the use of per-and polyfluoroalkyl substances (PFAS) in cosmetics.
MoCRA sets a 3-year deadline for the FDA to issue an assessment of the use and potential risks of PFAS in cosmetics products. PFAS are sometimes added as ingredients in some cosmetic products, including lotions, cleansers, nail polish, shaving cream, foundation, lipstick, eyeliner, eyeshadow, and mascara, according to the FDA. Sometimes the presence of PFAS in cosmetics is unintentional and is the result of impurities in raw materials or is due to the breakdown of ingredients, the FDA said.
The FDA’s website says that so far, the available research doesn’t allow for “definitive conclusions about the potential health risks of PFAS in cosmetics.”
The Centers for Disease Control and Prevention has stated that research has suggested potential links between high levels of certain PFAS, in general, with increased cholesterol levels, changes in liver enzyme levels, increased risk of hypertension or preeclampsia in pregnant women, and increased risk of kidney or testicular cancer.
PFAS compounds often are used to resist grease, oil, water, and heat in industrial settings. They are used in thousands of products, from nonstick cookware to firefighting foams and protective gear, because they can reduce friction, according to a National Academies of Sciences, Engineering, and Medicine report on PFAS that was issued last year.
PFAS are known as “forever chemicals” because they contain a carbon-fluorine bond, which does not break naturally. Even when PFAS are transformed in the body, they can assume other forms of PFAS that preserve the troublesome carbon-fluorine bond. With PFAS, the human body is confronted with a substance it doesn’t have the tools to process.
This is in contrast to proteins and carbohydrates, which are in a sense prepackaged for relatively easy disassembly in the human body. Many of these compounds have weak links that enzymes and stomach acid can take apart, such as sulfur-to-sulfur (disulfide) bonds. That’s why protein-based biotech drugs are injected instead of administered as pills. The ultimate goal of this digestion is for the body to gain energy from these compounds.
But with PFAS, the body faces the challenge of carbon-fluorine bonds that are very hard to break down, and there is no payoff for these efforts, Graham F. Peaslee, PhD, professor of physics at the University of Notre Dame (Indiana), told this news organization.
“Nothing will naturally eat it because when you break the bond, it’s like eating celery,” he said. “You use more calories to eat the celery than you gain back from it.”
Interest from a U.S. senator
Dr. Peaslee was one of the authors of a 2021 article about PFAS in cosmetics that appeared in the journal Environmental Science and Technology Letters.
In the article, Dr. Peaslee and colleagues reported on their screening of 231 cosmetic products purchased in the United States and Canada using particle-induced gamma-ray emission spectroscopy. They found cases of undisclosed PFAS in cosmetic products. Foundations, mascaras, and lip products were noted as being especially problematic.
Sen. Susan Collins (R-ME) cited Dr. Peaslee’s article in a 2021 floor speech as she argued for having the FDA ban the intentional addition of PFAS to cosmetics.
“The findings of this study are particularly alarming, as many of these products are subject to direct human exposure,” Sen. Collins said. “For example, lipstick is often inadvertently ingested, and mascara is sometimes absorbed through tear ducts.”
In addition, workers at cosmetics plants may be exposed to PFAS and discarded cosmetics that have these compounds, which could potentially contaminate drinking water, Sen. Collins said. In 2021, she introduced legislation seeking a ban on PFAS that are intentionally added to cosmetics. That legislation did not advance through the Senate.
But the Senate Appropriations Committee, on which Sen. Collins is the ranking Republican, wants the FDA to keep a ban on PFAS in mind.
The Senate Agriculture Appropriations subcommittee, which oversees the FDA’s budget, raised the issue of PFAS and cosmetics in a June report. The FDA should develop a plan outlining research needed to inform “regulatory decision making, including potential development of a proposed rule to ban intentionally added PFAS substances in cosmetics,” the subcommittee said.
A version of this article first appeared on Medscape.com.
They are also preparing to assess potential risks of so-called forever chemicals in these products.
The Food and Drug Administration last year gained new authority over cosmetics when Congress passed the Modernization of Cosmetics Regulation Act of 2022 (MoCRA) by adding this bill to a December budget package.
“On average, consumers in the U.S. use six to 12 cosmetics products daily. But, until recently the FDA didn’t have the authority to require manufacturers to submit cosmetic product listings, including a list of ingredients used in these products, or register the facilities where they were produced,” Namandjé Bumpus, PhD, FDA’s chief scientist, said in a press release.
In the statement, the FDA announced the release of a draft guidance document that is intended to help companies comply with the transparency requirements slated to kick in this December. The agency is accepting comments on this draft guidance through Sept. 7.
“Later this year, registration and listing of cosmetic product facilities and products will become a requirement, making information about cosmetic products, including the ingredients used in products and the facilities where they are produced, readily available to the agency,” Dr. Bumpus said.
The products, according to the FDA statement, include makeup, nail polishes, shaving creams, other grooming products, perfumes, face and body cleansers, hair products, moisturizers, and other skin care items.
MoCRA “represents a sea change in how FDA regulates the cosmetics industry,” attorneys Frederick R. Ball, Alyson Walker Lotman, and Kelly A. Bonner, wrote in an article for the Food and Drug Law Institute published in spring 2023.
The FDA has called the MoCRA law “the most significant expansion” of its authority to regulate cosmetics since the Federal Food, Drug, and Cosmetic Act was passed in 1938.
The agency is in the process of expanding its staff to carry out newly authorized duties, including the tracking of adverse events. The FDA budget request for fiscal 2024, which begins Oct. 1, seeks $5 million for work needed to implement MoCRA.
PFAS, or ‘forever chemicals’
Some of the requested FDA funding is intended to prepare the agency to assess the use of per-and polyfluoroalkyl substances (PFAS) in cosmetics.
MoCRA sets a 3-year deadline for the FDA to issue an assessment of the use and potential risks of PFAS in cosmetics products. PFAS are sometimes added as ingredients in some cosmetic products, including lotions, cleansers, nail polish, shaving cream, foundation, lipstick, eyeliner, eyeshadow, and mascara, according to the FDA. Sometimes the presence of PFAS in cosmetics is unintentional and is the result of impurities in raw materials or is due to the breakdown of ingredients, the FDA said.
The FDA’s website says that so far, the available research doesn’t allow for “definitive conclusions about the potential health risks of PFAS in cosmetics.”
The Centers for Disease Control and Prevention has stated that research has suggested potential links between high levels of certain PFAS, in general, with increased cholesterol levels, changes in liver enzyme levels, increased risk of hypertension or preeclampsia in pregnant women, and increased risk of kidney or testicular cancer.
PFAS compounds often are used to resist grease, oil, water, and heat in industrial settings. They are used in thousands of products, from nonstick cookware to firefighting foams and protective gear, because they can reduce friction, according to a National Academies of Sciences, Engineering, and Medicine report on PFAS that was issued last year.
PFAS are known as “forever chemicals” because they contain a carbon-fluorine bond, which does not break naturally. Even when PFAS are transformed in the body, they can assume other forms of PFAS that preserve the troublesome carbon-fluorine bond. With PFAS, the human body is confronted with a substance it doesn’t have the tools to process.
This is in contrast to proteins and carbohydrates, which are in a sense prepackaged for relatively easy disassembly in the human body. Many of these compounds have weak links that enzymes and stomach acid can take apart, such as sulfur-to-sulfur (disulfide) bonds. That’s why protein-based biotech drugs are injected instead of administered as pills. The ultimate goal of this digestion is for the body to gain energy from these compounds.
But with PFAS, the body faces the challenge of carbon-fluorine bonds that are very hard to break down, and there is no payoff for these efforts, Graham F. Peaslee, PhD, professor of physics at the University of Notre Dame (Indiana), told this news organization.
“Nothing will naturally eat it because when you break the bond, it’s like eating celery,” he said. “You use more calories to eat the celery than you gain back from it.”
Interest from a U.S. senator
Dr. Peaslee was one of the authors of a 2021 article about PFAS in cosmetics that appeared in the journal Environmental Science and Technology Letters.
In the article, Dr. Peaslee and colleagues reported on their screening of 231 cosmetic products purchased in the United States and Canada using particle-induced gamma-ray emission spectroscopy. They found cases of undisclosed PFAS in cosmetic products. Foundations, mascaras, and lip products were noted as being especially problematic.
Sen. Susan Collins (R-ME) cited Dr. Peaslee’s article in a 2021 floor speech as she argued for having the FDA ban the intentional addition of PFAS to cosmetics.
“The findings of this study are particularly alarming, as many of these products are subject to direct human exposure,” Sen. Collins said. “For example, lipstick is often inadvertently ingested, and mascara is sometimes absorbed through tear ducts.”
In addition, workers at cosmetics plants may be exposed to PFAS and discarded cosmetics that have these compounds, which could potentially contaminate drinking water, Sen. Collins said. In 2021, she introduced legislation seeking a ban on PFAS that are intentionally added to cosmetics. That legislation did not advance through the Senate.
But the Senate Appropriations Committee, on which Sen. Collins is the ranking Republican, wants the FDA to keep a ban on PFAS in mind.
The Senate Agriculture Appropriations subcommittee, which oversees the FDA’s budget, raised the issue of PFAS and cosmetics in a June report. The FDA should develop a plan outlining research needed to inform “regulatory decision making, including potential development of a proposed rule to ban intentionally added PFAS substances in cosmetics,” the subcommittee said.
A version of this article first appeared on Medscape.com.
FDA approves canakinumab for gout flares
The U.S. Food and Drug Administration has approved canakinumab (Ilaris) for the treatment of gout flares in adults who cannot be treated with NSAIDs, colchicine, or repeated courses of corticosteroids. The drug is also indicated for people who could not tolerate or had an inadequate response to NSAIDs or colchicine.
The drug, a humanized anti–interleukin-1 beta monoclonal antibody, is the first and only biologic approved in the United States for the treatment of gout flares, according to Novartis. It is administered in a single, subcutaneous injection of 150 mg.
“At Novartis, we are committed to bringing medicines that address high unmet needs to patients. We are proud to receive approval on our eighth indication for Ilaris in the U.S. and provide the first biologic medicine option for people with gout flares to help treat this painful and debilitating condition,” the company said in a statement to this news organization.
Canakinumab was first approved in the United States in 2009 for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS). Since then, it has been approved for the treatment of several other autoinflammatory diseases, including Still’s disease and recurrent fever syndromes.
In 2011, an FDA advisory panel voted against the approval of canakinumab to treat acute gout flares refractory to NSAIDs, colchicine, or repeated courses of corticosteroids, while in 2013, the European Medicine Agency approved the drug for this treatment indication.
Since that FDA advisory committee meeting and the FDA’s subsequent rejection letter, “[Novartis] has conducted additional studies in patients with gout flares and other related populations to further characterize the short- and long-term safety of canakinumab supporting the current application. To further support the benefit-risk [profile of the drug], the indication is for a more restricted population than initially proposed in 2011,” the FDA’s Center for Drug Evaluation and Research said in a statement to this news organization. “Given these considerations and the available safety information, the Agency determined that canakinumab, at the recommended dosage, has a favorable risk-benefit profile” in the specified patient population.
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration has approved canakinumab (Ilaris) for the treatment of gout flares in adults who cannot be treated with NSAIDs, colchicine, or repeated courses of corticosteroids. The drug is also indicated for people who could not tolerate or had an inadequate response to NSAIDs or colchicine.
The drug, a humanized anti–interleukin-1 beta monoclonal antibody, is the first and only biologic approved in the United States for the treatment of gout flares, according to Novartis. It is administered in a single, subcutaneous injection of 150 mg.
“At Novartis, we are committed to bringing medicines that address high unmet needs to patients. We are proud to receive approval on our eighth indication for Ilaris in the U.S. and provide the first biologic medicine option for people with gout flares to help treat this painful and debilitating condition,” the company said in a statement to this news organization.
Canakinumab was first approved in the United States in 2009 for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS). Since then, it has been approved for the treatment of several other autoinflammatory diseases, including Still’s disease and recurrent fever syndromes.
In 2011, an FDA advisory panel voted against the approval of canakinumab to treat acute gout flares refractory to NSAIDs, colchicine, or repeated courses of corticosteroids, while in 2013, the European Medicine Agency approved the drug for this treatment indication.
Since that FDA advisory committee meeting and the FDA’s subsequent rejection letter, “[Novartis] has conducted additional studies in patients with gout flares and other related populations to further characterize the short- and long-term safety of canakinumab supporting the current application. To further support the benefit-risk [profile of the drug], the indication is for a more restricted population than initially proposed in 2011,” the FDA’s Center for Drug Evaluation and Research said in a statement to this news organization. “Given these considerations and the available safety information, the Agency determined that canakinumab, at the recommended dosage, has a favorable risk-benefit profile” in the specified patient population.
A version of this article first appeared on Medscape.com.
The U.S. Food and Drug Administration has approved canakinumab (Ilaris) for the treatment of gout flares in adults who cannot be treated with NSAIDs, colchicine, or repeated courses of corticosteroids. The drug is also indicated for people who could not tolerate or had an inadequate response to NSAIDs or colchicine.
The drug, a humanized anti–interleukin-1 beta monoclonal antibody, is the first and only biologic approved in the United States for the treatment of gout flares, according to Novartis. It is administered in a single, subcutaneous injection of 150 mg.
“At Novartis, we are committed to bringing medicines that address high unmet needs to patients. We are proud to receive approval on our eighth indication for Ilaris in the U.S. and provide the first biologic medicine option for people with gout flares to help treat this painful and debilitating condition,” the company said in a statement to this news organization.
Canakinumab was first approved in the United States in 2009 for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS). Since then, it has been approved for the treatment of several other autoinflammatory diseases, including Still’s disease and recurrent fever syndromes.
In 2011, an FDA advisory panel voted against the approval of canakinumab to treat acute gout flares refractory to NSAIDs, colchicine, or repeated courses of corticosteroids, while in 2013, the European Medicine Agency approved the drug for this treatment indication.
Since that FDA advisory committee meeting and the FDA’s subsequent rejection letter, “[Novartis] has conducted additional studies in patients with gout flares and other related populations to further characterize the short- and long-term safety of canakinumab supporting the current application. To further support the benefit-risk [profile of the drug], the indication is for a more restricted population than initially proposed in 2011,” the FDA’s Center for Drug Evaluation and Research said in a statement to this news organization. “Given these considerations and the available safety information, the Agency determined that canakinumab, at the recommended dosage, has a favorable risk-benefit profile” in the specified patient population.
A version of this article first appeared on Medscape.com.
FDA clears new capabilities for diabetes app BlueStar
The latest clearance, announced on Aug. 23, enables the app-based platform to provide bolus insulin dose recommendations that are based on glucose and trend data from a compatible continuous glucose monitoring (CGM) device. On Aug. 15, the FDA cleared the BlueStar to use connected insulin dosing data in personalized bolus insulin dosing recommendations.
“Welldoc is the first company to receive clearance for a CGM-informed bolus calculator specifically designed for adults who manage their diabetes with multiple daily injections of insulin,” according to a company statement.
“With this clearance, Welldoc is filling a significant gap for people who require complex insulin regimens. By connecting directly with CGM data and using both glucose values and trend arrows, the BlueStar solution will provide precise and in-the-moment insulin dosing guidance directly to individuals, helping them reach their glucose targets,” endocrinologist Grazia Aleppo, MD, of Northwestern University, Chicago, said in the statement.
The new features extend the platform’s existing digital diet and lifestyle coaching capabilities. Previous FDA clearances included expansions to use most types of available insulins, including bolus and premixed insulin titration for patients with type 2 diabetes, in September 2021 and for basal insulin adjustment in June 2020.
Dr. Aleppo was a principal investigator in Welldoc’s clinical validation study for BlueStar.
A version of this article first appeared on Medscape.com.
The latest clearance, announced on Aug. 23, enables the app-based platform to provide bolus insulin dose recommendations that are based on glucose and trend data from a compatible continuous glucose monitoring (CGM) device. On Aug. 15, the FDA cleared the BlueStar to use connected insulin dosing data in personalized bolus insulin dosing recommendations.
“Welldoc is the first company to receive clearance for a CGM-informed bolus calculator specifically designed for adults who manage their diabetes with multiple daily injections of insulin,” according to a company statement.
“With this clearance, Welldoc is filling a significant gap for people who require complex insulin regimens. By connecting directly with CGM data and using both glucose values and trend arrows, the BlueStar solution will provide precise and in-the-moment insulin dosing guidance directly to individuals, helping them reach their glucose targets,” endocrinologist Grazia Aleppo, MD, of Northwestern University, Chicago, said in the statement.
The new features extend the platform’s existing digital diet and lifestyle coaching capabilities. Previous FDA clearances included expansions to use most types of available insulins, including bolus and premixed insulin titration for patients with type 2 diabetes, in September 2021 and for basal insulin adjustment in June 2020.
Dr. Aleppo was a principal investigator in Welldoc’s clinical validation study for BlueStar.
A version of this article first appeared on Medscape.com.
The latest clearance, announced on Aug. 23, enables the app-based platform to provide bolus insulin dose recommendations that are based on glucose and trend data from a compatible continuous glucose monitoring (CGM) device. On Aug. 15, the FDA cleared the BlueStar to use connected insulin dosing data in personalized bolus insulin dosing recommendations.
“Welldoc is the first company to receive clearance for a CGM-informed bolus calculator specifically designed for adults who manage their diabetes with multiple daily injections of insulin,” according to a company statement.
“With this clearance, Welldoc is filling a significant gap for people who require complex insulin regimens. By connecting directly with CGM data and using both glucose values and trend arrows, the BlueStar solution will provide precise and in-the-moment insulin dosing guidance directly to individuals, helping them reach their glucose targets,” endocrinologist Grazia Aleppo, MD, of Northwestern University, Chicago, said in the statement.
The new features extend the platform’s existing digital diet and lifestyle coaching capabilities. Previous FDA clearances included expansions to use most types of available insulins, including bolus and premixed insulin titration for patients with type 2 diabetes, in September 2021 and for basal insulin adjustment in June 2020.
Dr. Aleppo was a principal investigator in Welldoc’s clinical validation study for BlueStar.
A version of this article first appeared on Medscape.com.
FDA panel split on efficacy of Spyral renal denervation system
The Food and Drug Administration’s Circulatory System Devices Panel unanimously agreed that the Symplicity Spyral Renal Denervation System (Medtronic) is safe, but the panel was split on its efficacy and whether the benefits outweighed the risks associated with its use.
The moderator’s tiebreaker vote went against the benefit-risk profile, for a final vote of 6 yes, 7 no, and 1 abstention.
The Symplicity Spyral system provides a catheter-based approach to denervate the renal arteries using radiofrequency energy. The proposed indication is for reduction of blood pressure in patients with uncontrolled hypertension despite their use of antihypertensive medications, or in patients who cannot tolerate antihypertensive medications.
The Spyral device received breakthrough device designation in March 2020. The FDA determined that the device met the criteria for inclusion in the program because it was a novel technology and had the potential to provide more effective treatment for patients with resistant or uncontrolled hypertension.
Medtronic presented data from two studies, the SPYRAL HTN-OFF and SPYRAL HTN-ON randomized trials.
HTN-OFF enrolled patients with hypertension whose medications could be discontinued at the start of the trial. The primary effectiveness endpoint was the mean difference in the baseline adjusted 24-hour ambulatory systolic blood pressure (ASBP) from baseline to 3 months post renal denervation or sham procedure. The study showed a statistically significant reduction of 3.9 mm Hg ASBP in patients who received the device compared with sham control patients.
HTN-ON evaluated patients with uncontrolled hypertension who continued taking their blood pressure medications during treatment with either the Spyral renal denervation device or a sham device. The primary endpoint was the mean difference in the baseline adjusted 24 hour ambulatory systolic blood pressure at 6 months. The study showed a nonsignificant 24-hour 0.03–mm Hg reduction in ambulatory systolic blood pressure in active-treatment patients compared with sham control patients.
Many on the panel agreed that the device was safe and effective. Randall Starling, MD, professor of medicine in the Heart, Vascular, and Thoracic Institute at Cleveland Clinic, said that he was comfortable with the data presented by Medtronic and that his affirmative vote reflected his recognition that hypertension is not effectively treated with current medications and that another tool in the armamentarium to treat patients is needed.
Matthew Corriere, MD, Frankel Professor of Cardiovascular Surgery at the University of Michigan, Ann Arbor, abstained from voting on whether the benefits of the system outweighed its risks. “I think there is potential benefit, but we don’t know which patients are most likely to have a benefit that outweighs any risks. More selective indications for this product could potentially tip the balance of the benefit outweighing the risks,” he said.
Robert Yeh, MD, director, Center for Outcomes Research in Cardiology, Beth Israel Deaconess Medical Center, Boston, said he believed that the device was safe and effective and that its use resulted in a favorable risk-benefit ratio for patients. He pointed to the wide variability in effectiveness across the patient population and suggested that as the device becomes more widely used, experience will show which patients will benefit the most from its use.
Keith Allen, MD, director of surgical research at St. Luke’s Hospital of Kansas City, Kansas City, Mo., said the data presented by Medtronic reassured him that the device was safe, but he said he remained unconvinced that the device was effective. “I think that, while this is a safe procedure, the efficacy was mild at best, and that was only at 3 months,” he said.
Other panel members agreed.
“Yes as to safety, but no as to effectiveness,” said Mark Lockhart, MD, professor, department of radiology, University of Alabama, Birmingham. “There is too much uncertainty about there actually being a real benefit to outweigh even a small risk of an invasive procedure,” he said.
One of the statisticians on the panel, Benjamin Saville, PhD, director and senior statistical scientist, Berry Consultants, Austin, Texas, said he did not feel that effectiveness was adequately demonstrated in the trial data presented by Medtronic.
He agreed there is a small but potentially clinically meaningful benefit but voted no because he did not think benefit was demonstrated for those patients in the proposed indication. “For me, I think I would need additional randomized data to convince me that the benefits outweigh the risks.”
Julia Lewis, MD, professor of medicine at Vanderbilt University, Nashville, Tenn., voted against endorsing the device for efficacy. “We have one study that is negative and one that is minimally positive,” and there is no reason to think one of those results is more valid than the other, she said.
“So as far as I’m concerned, we still don’t know the efficacy of this, and if it gets on the market, the anecdotal, small sample size of each individual physician using this intervention will not allow them to select out the patients that will benefit from those who won’t benefit, and to not have a definitive study that better defines that it is efficacious and in whom is actually a disservice to the public,” she concluded.
After the panel meeting, Medtronic issued a statement on the result.
“We appreciate the robust conversation that occurred prior to the vote,” Jason Weidman, senior vice-president of the coronary and renal denervation business at Medtronic, said in the statement. “We will continue to collaborate with the FDA on bringing a new option to the millions of people living with high blood pressure.”
The lead investigator of the SPYRAL HTN-ON MED trial, David Kandzari, MD, chief at Piedmont Heart Institute and Cardiovascular Services, added, “The totality of the evidence demonstrated that there is a benefit with the SPYRAL RDN catheter, and there is no question about the safety of the procedure.”
A version of this article first appeared on Medscape.com.
The Food and Drug Administration’s Circulatory System Devices Panel unanimously agreed that the Symplicity Spyral Renal Denervation System (Medtronic) is safe, but the panel was split on its efficacy and whether the benefits outweighed the risks associated with its use.
The moderator’s tiebreaker vote went against the benefit-risk profile, for a final vote of 6 yes, 7 no, and 1 abstention.
The Symplicity Spyral system provides a catheter-based approach to denervate the renal arteries using radiofrequency energy. The proposed indication is for reduction of blood pressure in patients with uncontrolled hypertension despite their use of antihypertensive medications, or in patients who cannot tolerate antihypertensive medications.
The Spyral device received breakthrough device designation in March 2020. The FDA determined that the device met the criteria for inclusion in the program because it was a novel technology and had the potential to provide more effective treatment for patients with resistant or uncontrolled hypertension.
Medtronic presented data from two studies, the SPYRAL HTN-OFF and SPYRAL HTN-ON randomized trials.
HTN-OFF enrolled patients with hypertension whose medications could be discontinued at the start of the trial. The primary effectiveness endpoint was the mean difference in the baseline adjusted 24-hour ambulatory systolic blood pressure (ASBP) from baseline to 3 months post renal denervation or sham procedure. The study showed a statistically significant reduction of 3.9 mm Hg ASBP in patients who received the device compared with sham control patients.
HTN-ON evaluated patients with uncontrolled hypertension who continued taking their blood pressure medications during treatment with either the Spyral renal denervation device or a sham device. The primary endpoint was the mean difference in the baseline adjusted 24 hour ambulatory systolic blood pressure at 6 months. The study showed a nonsignificant 24-hour 0.03–mm Hg reduction in ambulatory systolic blood pressure in active-treatment patients compared with sham control patients.
Many on the panel agreed that the device was safe and effective. Randall Starling, MD, professor of medicine in the Heart, Vascular, and Thoracic Institute at Cleveland Clinic, said that he was comfortable with the data presented by Medtronic and that his affirmative vote reflected his recognition that hypertension is not effectively treated with current medications and that another tool in the armamentarium to treat patients is needed.
Matthew Corriere, MD, Frankel Professor of Cardiovascular Surgery at the University of Michigan, Ann Arbor, abstained from voting on whether the benefits of the system outweighed its risks. “I think there is potential benefit, but we don’t know which patients are most likely to have a benefit that outweighs any risks. More selective indications for this product could potentially tip the balance of the benefit outweighing the risks,” he said.
Robert Yeh, MD, director, Center for Outcomes Research in Cardiology, Beth Israel Deaconess Medical Center, Boston, said he believed that the device was safe and effective and that its use resulted in a favorable risk-benefit ratio for patients. He pointed to the wide variability in effectiveness across the patient population and suggested that as the device becomes more widely used, experience will show which patients will benefit the most from its use.
Keith Allen, MD, director of surgical research at St. Luke’s Hospital of Kansas City, Kansas City, Mo., said the data presented by Medtronic reassured him that the device was safe, but he said he remained unconvinced that the device was effective. “I think that, while this is a safe procedure, the efficacy was mild at best, and that was only at 3 months,” he said.
Other panel members agreed.
“Yes as to safety, but no as to effectiveness,” said Mark Lockhart, MD, professor, department of radiology, University of Alabama, Birmingham. “There is too much uncertainty about there actually being a real benefit to outweigh even a small risk of an invasive procedure,” he said.
One of the statisticians on the panel, Benjamin Saville, PhD, director and senior statistical scientist, Berry Consultants, Austin, Texas, said he did not feel that effectiveness was adequately demonstrated in the trial data presented by Medtronic.
He agreed there is a small but potentially clinically meaningful benefit but voted no because he did not think benefit was demonstrated for those patients in the proposed indication. “For me, I think I would need additional randomized data to convince me that the benefits outweigh the risks.”
Julia Lewis, MD, professor of medicine at Vanderbilt University, Nashville, Tenn., voted against endorsing the device for efficacy. “We have one study that is negative and one that is minimally positive,” and there is no reason to think one of those results is more valid than the other, she said.
“So as far as I’m concerned, we still don’t know the efficacy of this, and if it gets on the market, the anecdotal, small sample size of each individual physician using this intervention will not allow them to select out the patients that will benefit from those who won’t benefit, and to not have a definitive study that better defines that it is efficacious and in whom is actually a disservice to the public,” she concluded.
After the panel meeting, Medtronic issued a statement on the result.
“We appreciate the robust conversation that occurred prior to the vote,” Jason Weidman, senior vice-president of the coronary and renal denervation business at Medtronic, said in the statement. “We will continue to collaborate with the FDA on bringing a new option to the millions of people living with high blood pressure.”
The lead investigator of the SPYRAL HTN-ON MED trial, David Kandzari, MD, chief at Piedmont Heart Institute and Cardiovascular Services, added, “The totality of the evidence demonstrated that there is a benefit with the SPYRAL RDN catheter, and there is no question about the safety of the procedure.”
A version of this article first appeared on Medscape.com.
The Food and Drug Administration’s Circulatory System Devices Panel unanimously agreed that the Symplicity Spyral Renal Denervation System (Medtronic) is safe, but the panel was split on its efficacy and whether the benefits outweighed the risks associated with its use.
The moderator’s tiebreaker vote went against the benefit-risk profile, for a final vote of 6 yes, 7 no, and 1 abstention.
The Symplicity Spyral system provides a catheter-based approach to denervate the renal arteries using radiofrequency energy. The proposed indication is for reduction of blood pressure in patients with uncontrolled hypertension despite their use of antihypertensive medications, or in patients who cannot tolerate antihypertensive medications.
The Spyral device received breakthrough device designation in March 2020. The FDA determined that the device met the criteria for inclusion in the program because it was a novel technology and had the potential to provide more effective treatment for patients with resistant or uncontrolled hypertension.
Medtronic presented data from two studies, the SPYRAL HTN-OFF and SPYRAL HTN-ON randomized trials.
HTN-OFF enrolled patients with hypertension whose medications could be discontinued at the start of the trial. The primary effectiveness endpoint was the mean difference in the baseline adjusted 24-hour ambulatory systolic blood pressure (ASBP) from baseline to 3 months post renal denervation or sham procedure. The study showed a statistically significant reduction of 3.9 mm Hg ASBP in patients who received the device compared with sham control patients.
HTN-ON evaluated patients with uncontrolled hypertension who continued taking their blood pressure medications during treatment with either the Spyral renal denervation device or a sham device. The primary endpoint was the mean difference in the baseline adjusted 24 hour ambulatory systolic blood pressure at 6 months. The study showed a nonsignificant 24-hour 0.03–mm Hg reduction in ambulatory systolic blood pressure in active-treatment patients compared with sham control patients.
Many on the panel agreed that the device was safe and effective. Randall Starling, MD, professor of medicine in the Heart, Vascular, and Thoracic Institute at Cleveland Clinic, said that he was comfortable with the data presented by Medtronic and that his affirmative vote reflected his recognition that hypertension is not effectively treated with current medications and that another tool in the armamentarium to treat patients is needed.
Matthew Corriere, MD, Frankel Professor of Cardiovascular Surgery at the University of Michigan, Ann Arbor, abstained from voting on whether the benefits of the system outweighed its risks. “I think there is potential benefit, but we don’t know which patients are most likely to have a benefit that outweighs any risks. More selective indications for this product could potentially tip the balance of the benefit outweighing the risks,” he said.
Robert Yeh, MD, director, Center for Outcomes Research in Cardiology, Beth Israel Deaconess Medical Center, Boston, said he believed that the device was safe and effective and that its use resulted in a favorable risk-benefit ratio for patients. He pointed to the wide variability in effectiveness across the patient population and suggested that as the device becomes more widely used, experience will show which patients will benefit the most from its use.
Keith Allen, MD, director of surgical research at St. Luke’s Hospital of Kansas City, Kansas City, Mo., said the data presented by Medtronic reassured him that the device was safe, but he said he remained unconvinced that the device was effective. “I think that, while this is a safe procedure, the efficacy was mild at best, and that was only at 3 months,” he said.
Other panel members agreed.
“Yes as to safety, but no as to effectiveness,” said Mark Lockhart, MD, professor, department of radiology, University of Alabama, Birmingham. “There is too much uncertainty about there actually being a real benefit to outweigh even a small risk of an invasive procedure,” he said.
One of the statisticians on the panel, Benjamin Saville, PhD, director and senior statistical scientist, Berry Consultants, Austin, Texas, said he did not feel that effectiveness was adequately demonstrated in the trial data presented by Medtronic.
He agreed there is a small but potentially clinically meaningful benefit but voted no because he did not think benefit was demonstrated for those patients in the proposed indication. “For me, I think I would need additional randomized data to convince me that the benefits outweigh the risks.”
Julia Lewis, MD, professor of medicine at Vanderbilt University, Nashville, Tenn., voted against endorsing the device for efficacy. “We have one study that is negative and one that is minimally positive,” and there is no reason to think one of those results is more valid than the other, she said.
“So as far as I’m concerned, we still don’t know the efficacy of this, and if it gets on the market, the anecdotal, small sample size of each individual physician using this intervention will not allow them to select out the patients that will benefit from those who won’t benefit, and to not have a definitive study that better defines that it is efficacious and in whom is actually a disservice to the public,” she concluded.
After the panel meeting, Medtronic issued a statement on the result.
“We appreciate the robust conversation that occurred prior to the vote,” Jason Weidman, senior vice-president of the coronary and renal denervation business at Medtronic, said in the statement. “We will continue to collaborate with the FDA on bringing a new option to the millions of people living with high blood pressure.”
The lead investigator of the SPYRAL HTN-ON MED trial, David Kandzari, MD, chief at Piedmont Heart Institute and Cardiovascular Services, added, “The totality of the evidence demonstrated that there is a benefit with the SPYRAL RDN catheter, and there is no question about the safety of the procedure.”
A version of this article first appeared on Medscape.com.
FDA approves new tubeless insulin pump
The product received CE Mark in Europe in 2018 and is now available in 19 markets worldwide. It offers users a choice of bolusing directly from the pump or from a handheld remote-control device. The pump can be detached and reattached without wasting insulin.
The remote-control device also incorporates blood glucose monitoring and bolus advice, although it currently does not integrate with continuous glucose monitoring (CGM) devices.
A Roche spokesperson said in an interview, “For future product generations, we are exploring possibilities to integrate CGM data into the system. Already today, the diabetes manager allows users to manually enter a glucose value that can be used to calculate a bolus. To do so, people with diabetes could use their CGM device of choice in conjunction with the Accu-Chek Solo micropump system.”
Roche will provide an update on next steps for further developments and time lines for launch “in due course,” according to a company statement.
A version of this article appeared on Medscape.com.
The product received CE Mark in Europe in 2018 and is now available in 19 markets worldwide. It offers users a choice of bolusing directly from the pump or from a handheld remote-control device. The pump can be detached and reattached without wasting insulin.
The remote-control device also incorporates blood glucose monitoring and bolus advice, although it currently does not integrate with continuous glucose monitoring (CGM) devices.
A Roche spokesperson said in an interview, “For future product generations, we are exploring possibilities to integrate CGM data into the system. Already today, the diabetes manager allows users to manually enter a glucose value that can be used to calculate a bolus. To do so, people with diabetes could use their CGM device of choice in conjunction with the Accu-Chek Solo micropump system.”
Roche will provide an update on next steps for further developments and time lines for launch “in due course,” according to a company statement.
A version of this article appeared on Medscape.com.
The product received CE Mark in Europe in 2018 and is now available in 19 markets worldwide. It offers users a choice of bolusing directly from the pump or from a handheld remote-control device. The pump can be detached and reattached without wasting insulin.
The remote-control device also incorporates blood glucose monitoring and bolus advice, although it currently does not integrate with continuous glucose monitoring (CGM) devices.
A Roche spokesperson said in an interview, “For future product generations, we are exploring possibilities to integrate CGM data into the system. Already today, the diabetes manager allows users to manually enter a glucose value that can be used to calculate a bolus. To do so, people with diabetes could use their CGM device of choice in conjunction with the Accu-Chek Solo micropump system.”
Roche will provide an update on next steps for further developments and time lines for launch “in due course,” according to a company statement.
A version of this article appeared on Medscape.com.
One in five women report mistreatment during maternity care
“We have to do better at providing respectful and unbiased care to all mothers,” Debra E. Houry, MD, chief medical officer of the Centers for Disease Control and Prevention, said in a press briefing announcing the findings, which were published as a Vital Signs report in the CDC’s Morbidity and Mortality Weekly Report.
Previous research showed an increase in maternal deaths in the United States from 17.4 to 32.9 per 100,000 live births between 2018 and 2021, but approximately 80% of these deaths are preventable, wrote Yousra A. Mohamoud, PhD, of the CDC’s division of reproductive health, and colleagues.
“Maternal mortality review committees have identified discrimination as one factor contributing to pregnancy-related deaths,” the researchers wrote. Respectful care must be part of a larger strategy to prevent these deaths, they emphasized.
In the report, researchers reviewed data from 2,402 women who responded to an opt-in survey. The survey was conducted for the CDC through Porter Novelli, and no personally identifying information was included. Nearly 70% of the participants were White, 10.7% were Black, 10.2% were Hispanic, 4.8% were Asian, 1.5% were American Indian, Alaska Native, Pacific Islander, or Native Hawaiian, 2.8% were multiracial, and 0.5% were another race.
The survey included questions about maternity care experiences during pregnancy and delivery of the youngest child. For 65.5% of respondents, their youngest child was 5 years or older at the time of the survey.
Mistreatment during maternity care was defined using seven validated questions, including questions about violations of physical privacy, verbal abuse, and inattention to requests for help. Satisfaction with maternity care was defined as “very satisfied” or “somewhat satisfied.”
Participants also responded to questions about discrimination during maternity care based on factors such as race, ethnicity, skin color, age, and weight. Finally, participants were asked whether they refrained from asking questions about their health or raising concerns with health care providers.
Overall, 20.4% of respondents reported experiencing one of the defined forms of mistreatment during maternity care. The most common mistreatment reported by the women was being ignored by providers when they requested help (9.7%), followed by being shouted at or scolded (6.7%), having physical privacy violated (5.1%), and being forced to accept unwanted treatment or threatened with withholding of treatment (4.6%).
However, approximately 90% of women overall and 75% of those who reported any mistreatment were very or somewhat satisfied with their maternity care.
When stratified by race, mistreatment was reported most frequently by Black, Hispanic, and multiracial women (30%, 29%, and 27%, respectively).
Overall, 29% of women reported experiencing some type of discrimination; the most frequently reported reasons were age, weight, and income. Black women reported the highest rates of discrimination (40%) followed by multiracial women (39%) and Hispanic women (37%).
With regard to self-advocacy, 45% of women reported holding back from asking questions of health care providers; the most common reasons were thinking their health concerns were normal for pregnancy, being embarrassed, and being concerned that health care providers would consider them difficult.
In addition, more women with no insurance or public insurance at the time of delivery reported mistreatment during their maternity care than did women with private insurance (28%, 26%, and 16%, respectively).
The findings were limited by several factors, including the opt-in nature of the survey, which means that the data are likely not representative of the birthing population in the United States, the researchers noted. Other limitations included the reliance on self-reports, potential recall bias, use of English language only, and use of a combined category for respondents of American Indian, Alaska Native, Native Hawaiian, and Pacific Islander ethnicity.
However, the results highlight the need for improving respectful care as part of a larger strategy to reduce pregnancy-related deaths, the researchers said. At the system level, quality improvement programs are needed to standardize care and support providers in recognizing and reducing biases and increasing cultural awareness and communication. At the provider level, clinicians at all points in the maternity care process can improve patient experiences by providing equitable and respectful care, and by listening to and addressing patients’ concerns.
In addition, communication campaigns and community engagement can include perspectives of patients, families, and communities to support women and encourage them to ask questions and express concerns, the researchers said.
Improving respectful care can be part of actions to reduce mortality at all levels, the researchers noted. The Hear Her campaign, developed by the CDC Foundation with funding from Merck, provides resources for pregnant and postpartum women and their support networks to help reduce pregnancy-related deaths and complications by encouraging women to share concerns with providers and to recognize urgent maternal warning signs.
The study received no outside funding. The researchers had no financial conflicts to disclose.
“We have to do better at providing respectful and unbiased care to all mothers,” Debra E. Houry, MD, chief medical officer of the Centers for Disease Control and Prevention, said in a press briefing announcing the findings, which were published as a Vital Signs report in the CDC’s Morbidity and Mortality Weekly Report.
Previous research showed an increase in maternal deaths in the United States from 17.4 to 32.9 per 100,000 live births between 2018 and 2021, but approximately 80% of these deaths are preventable, wrote Yousra A. Mohamoud, PhD, of the CDC’s division of reproductive health, and colleagues.
“Maternal mortality review committees have identified discrimination as one factor contributing to pregnancy-related deaths,” the researchers wrote. Respectful care must be part of a larger strategy to prevent these deaths, they emphasized.
In the report, researchers reviewed data from 2,402 women who responded to an opt-in survey. The survey was conducted for the CDC through Porter Novelli, and no personally identifying information was included. Nearly 70% of the participants were White, 10.7% were Black, 10.2% were Hispanic, 4.8% were Asian, 1.5% were American Indian, Alaska Native, Pacific Islander, or Native Hawaiian, 2.8% were multiracial, and 0.5% were another race.
The survey included questions about maternity care experiences during pregnancy and delivery of the youngest child. For 65.5% of respondents, their youngest child was 5 years or older at the time of the survey.
Mistreatment during maternity care was defined using seven validated questions, including questions about violations of physical privacy, verbal abuse, and inattention to requests for help. Satisfaction with maternity care was defined as “very satisfied” or “somewhat satisfied.”
Participants also responded to questions about discrimination during maternity care based on factors such as race, ethnicity, skin color, age, and weight. Finally, participants were asked whether they refrained from asking questions about their health or raising concerns with health care providers.
Overall, 20.4% of respondents reported experiencing one of the defined forms of mistreatment during maternity care. The most common mistreatment reported by the women was being ignored by providers when they requested help (9.7%), followed by being shouted at or scolded (6.7%), having physical privacy violated (5.1%), and being forced to accept unwanted treatment or threatened with withholding of treatment (4.6%).
However, approximately 90% of women overall and 75% of those who reported any mistreatment were very or somewhat satisfied with their maternity care.
When stratified by race, mistreatment was reported most frequently by Black, Hispanic, and multiracial women (30%, 29%, and 27%, respectively).
Overall, 29% of women reported experiencing some type of discrimination; the most frequently reported reasons were age, weight, and income. Black women reported the highest rates of discrimination (40%) followed by multiracial women (39%) and Hispanic women (37%).
With regard to self-advocacy, 45% of women reported holding back from asking questions of health care providers; the most common reasons were thinking their health concerns were normal for pregnancy, being embarrassed, and being concerned that health care providers would consider them difficult.
In addition, more women with no insurance or public insurance at the time of delivery reported mistreatment during their maternity care than did women with private insurance (28%, 26%, and 16%, respectively).
The findings were limited by several factors, including the opt-in nature of the survey, which means that the data are likely not representative of the birthing population in the United States, the researchers noted. Other limitations included the reliance on self-reports, potential recall bias, use of English language only, and use of a combined category for respondents of American Indian, Alaska Native, Native Hawaiian, and Pacific Islander ethnicity.
However, the results highlight the need for improving respectful care as part of a larger strategy to reduce pregnancy-related deaths, the researchers said. At the system level, quality improvement programs are needed to standardize care and support providers in recognizing and reducing biases and increasing cultural awareness and communication. At the provider level, clinicians at all points in the maternity care process can improve patient experiences by providing equitable and respectful care, and by listening to and addressing patients’ concerns.
In addition, communication campaigns and community engagement can include perspectives of patients, families, and communities to support women and encourage them to ask questions and express concerns, the researchers said.
Improving respectful care can be part of actions to reduce mortality at all levels, the researchers noted. The Hear Her campaign, developed by the CDC Foundation with funding from Merck, provides resources for pregnant and postpartum women and their support networks to help reduce pregnancy-related deaths and complications by encouraging women to share concerns with providers and to recognize urgent maternal warning signs.
The study received no outside funding. The researchers had no financial conflicts to disclose.
“We have to do better at providing respectful and unbiased care to all mothers,” Debra E. Houry, MD, chief medical officer of the Centers for Disease Control and Prevention, said in a press briefing announcing the findings, which were published as a Vital Signs report in the CDC’s Morbidity and Mortality Weekly Report.
Previous research showed an increase in maternal deaths in the United States from 17.4 to 32.9 per 100,000 live births between 2018 and 2021, but approximately 80% of these deaths are preventable, wrote Yousra A. Mohamoud, PhD, of the CDC’s division of reproductive health, and colleagues.
“Maternal mortality review committees have identified discrimination as one factor contributing to pregnancy-related deaths,” the researchers wrote. Respectful care must be part of a larger strategy to prevent these deaths, they emphasized.
In the report, researchers reviewed data from 2,402 women who responded to an opt-in survey. The survey was conducted for the CDC through Porter Novelli, and no personally identifying information was included. Nearly 70% of the participants were White, 10.7% were Black, 10.2% were Hispanic, 4.8% were Asian, 1.5% were American Indian, Alaska Native, Pacific Islander, or Native Hawaiian, 2.8% were multiracial, and 0.5% were another race.
The survey included questions about maternity care experiences during pregnancy and delivery of the youngest child. For 65.5% of respondents, their youngest child was 5 years or older at the time of the survey.
Mistreatment during maternity care was defined using seven validated questions, including questions about violations of physical privacy, verbal abuse, and inattention to requests for help. Satisfaction with maternity care was defined as “very satisfied” or “somewhat satisfied.”
Participants also responded to questions about discrimination during maternity care based on factors such as race, ethnicity, skin color, age, and weight. Finally, participants were asked whether they refrained from asking questions about their health or raising concerns with health care providers.
Overall, 20.4% of respondents reported experiencing one of the defined forms of mistreatment during maternity care. The most common mistreatment reported by the women was being ignored by providers when they requested help (9.7%), followed by being shouted at or scolded (6.7%), having physical privacy violated (5.1%), and being forced to accept unwanted treatment or threatened with withholding of treatment (4.6%).
However, approximately 90% of women overall and 75% of those who reported any mistreatment were very or somewhat satisfied with their maternity care.
When stratified by race, mistreatment was reported most frequently by Black, Hispanic, and multiracial women (30%, 29%, and 27%, respectively).
Overall, 29% of women reported experiencing some type of discrimination; the most frequently reported reasons were age, weight, and income. Black women reported the highest rates of discrimination (40%) followed by multiracial women (39%) and Hispanic women (37%).
With regard to self-advocacy, 45% of women reported holding back from asking questions of health care providers; the most common reasons were thinking their health concerns were normal for pregnancy, being embarrassed, and being concerned that health care providers would consider them difficult.
In addition, more women with no insurance or public insurance at the time of delivery reported mistreatment during their maternity care than did women with private insurance (28%, 26%, and 16%, respectively).
The findings were limited by several factors, including the opt-in nature of the survey, which means that the data are likely not representative of the birthing population in the United States, the researchers noted. Other limitations included the reliance on self-reports, potential recall bias, use of English language only, and use of a combined category for respondents of American Indian, Alaska Native, Native Hawaiian, and Pacific Islander ethnicity.
However, the results highlight the need for improving respectful care as part of a larger strategy to reduce pregnancy-related deaths, the researchers said. At the system level, quality improvement programs are needed to standardize care and support providers in recognizing and reducing biases and increasing cultural awareness and communication. At the provider level, clinicians at all points in the maternity care process can improve patient experiences by providing equitable and respectful care, and by listening to and addressing patients’ concerns.
In addition, communication campaigns and community engagement can include perspectives of patients, families, and communities to support women and encourage them to ask questions and express concerns, the researchers said.
Improving respectful care can be part of actions to reduce mortality at all levels, the researchers noted. The Hear Her campaign, developed by the CDC Foundation with funding from Merck, provides resources for pregnant and postpartum women and their support networks to help reduce pregnancy-related deaths and complications by encouraging women to share concerns with providers and to recognize urgent maternal warning signs.
The study received no outside funding. The researchers had no financial conflicts to disclose.
FROM MMWR
ReCor renal denervation system safe, effective: FDA panel
in lowering blood pressure for adults with uncontrolled hypertension who may be inadequately responsive to, or who are intolerant of, antihypertensive medications.
The device is intended to be used in renal arteries with diameters of 3.0 to 8.0 mm.
After hearing data from three trials, RADIANCE-HTN SOLO, RADIANCE II, and RADIANCE-HTN TRIO, the 12-member panel unanimously agreed that there was “reasonable assurance” that the ReCor Paradise Ultrasound Renal Denervation System (ReCor Medical) was safe.
However, while most of the panel felt the device was effective, a few disagreed.
Keith Allen, MD, director of surgical research for the Mid-America Heart and Lung Surgeons, Kansas City, Mo., who was one of the three panel members who voted no regarding efficacy, stated that he had concerns about the duration and the degree of efficacy shown in the trials.
Mark Lockhart, MD, University of Alabama, Birmingham, also voted no. “I do think there was an effect for 2 months, but the duration of that positive effect appears to decline after that period of time.”
Benjamin Saville, PhD, echoed Dr. Lockhart’s concern: “The benefit is more short term, it is unclear what the long term benefit would be.”
Data from all three trials showed a significant drop in blood pressure with the device compared with pharmacologic therapy, but after 2 months of follow-up, that advantage disappeared.
The FDA highlighted data from the trials that showed that at 2 months, uRDN patients experienced significant reductions in systolic blood pressure compared with those treated with a sham device; however, by 6 months, there was a difference of only 1 mm Hg between the two groups.
“It seems when I look at 6 months and 12 months, the benefit is very tiny. We know the safety is fine, but a benefit of less than 1 mm Hg difference would not make me want to have an intervention,” said statistician Janet Wittes, PhD.
“I think the device is efficacious, even though there is not much difference between sham and treatment, but a big issue is the fact that half of our patients are not compliant. That will make the benefits over sham more clear,” noted Jim Blankenship, MD, professor of medicine and director of the division of cardiology at the University of New Mexico in Albuquerque.
John Hirshfeld Jr., MD, professor emeritus of medicine at the University of Pennsylvania, Philadelphia, said he voted yes on safety and efficacy but admitted he had some misgivings. “The sample size was small, but it is a novel tool to add to our tool box, and hopefully it will be used responsibly,” he said.
John Somberg, MD, professor emeritus of medicine, cardiology, and pharmacology, Rush University, Chicago, said the data on this procedure show “that antihypertensive medication works. Denervation is not superior to medications. It lowers blood pressure and is persistent, but when you can take the sham group to almost as good control as you get in the denervation group, that shows it can also be done with medicines.”
The panel wants to see results from additional studies in important subpopulations who are affected by hypertension, including Black people, women, the elderly, and people who already have cardiovascular risk factors, such as diabetes and heart failure.
Deneen Hesser, RN, the panel’s patient representative, called for any postmarketing studies that may be conducted by ReCor to include a good patient education program and also a way of documenting patient-reported outcomes.
“This would help us ascertain how happy people were if they were able to reduce their medication burden by, for example, one drug, or if they were willing to undergo a procedure to be able to stop taking so many antihypertensive medications,” she said.
The panel will meet again to review data on Medtronic’s Simplicity Spyral Renal Denervation System, which is also for patients with uncontrolled hypertension.
A version of this article appeared on Medscape.com.
in lowering blood pressure for adults with uncontrolled hypertension who may be inadequately responsive to, or who are intolerant of, antihypertensive medications.
The device is intended to be used in renal arteries with diameters of 3.0 to 8.0 mm.
After hearing data from three trials, RADIANCE-HTN SOLO, RADIANCE II, and RADIANCE-HTN TRIO, the 12-member panel unanimously agreed that there was “reasonable assurance” that the ReCor Paradise Ultrasound Renal Denervation System (ReCor Medical) was safe.
However, while most of the panel felt the device was effective, a few disagreed.
Keith Allen, MD, director of surgical research for the Mid-America Heart and Lung Surgeons, Kansas City, Mo., who was one of the three panel members who voted no regarding efficacy, stated that he had concerns about the duration and the degree of efficacy shown in the trials.
Mark Lockhart, MD, University of Alabama, Birmingham, also voted no. “I do think there was an effect for 2 months, but the duration of that positive effect appears to decline after that period of time.”
Benjamin Saville, PhD, echoed Dr. Lockhart’s concern: “The benefit is more short term, it is unclear what the long term benefit would be.”
Data from all three trials showed a significant drop in blood pressure with the device compared with pharmacologic therapy, but after 2 months of follow-up, that advantage disappeared.
The FDA highlighted data from the trials that showed that at 2 months, uRDN patients experienced significant reductions in systolic blood pressure compared with those treated with a sham device; however, by 6 months, there was a difference of only 1 mm Hg between the two groups.
“It seems when I look at 6 months and 12 months, the benefit is very tiny. We know the safety is fine, but a benefit of less than 1 mm Hg difference would not make me want to have an intervention,” said statistician Janet Wittes, PhD.
“I think the device is efficacious, even though there is not much difference between sham and treatment, but a big issue is the fact that half of our patients are not compliant. That will make the benefits over sham more clear,” noted Jim Blankenship, MD, professor of medicine and director of the division of cardiology at the University of New Mexico in Albuquerque.
John Hirshfeld Jr., MD, professor emeritus of medicine at the University of Pennsylvania, Philadelphia, said he voted yes on safety and efficacy but admitted he had some misgivings. “The sample size was small, but it is a novel tool to add to our tool box, and hopefully it will be used responsibly,” he said.
John Somberg, MD, professor emeritus of medicine, cardiology, and pharmacology, Rush University, Chicago, said the data on this procedure show “that antihypertensive medication works. Denervation is not superior to medications. It lowers blood pressure and is persistent, but when you can take the sham group to almost as good control as you get in the denervation group, that shows it can also be done with medicines.”
The panel wants to see results from additional studies in important subpopulations who are affected by hypertension, including Black people, women, the elderly, and people who already have cardiovascular risk factors, such as diabetes and heart failure.
Deneen Hesser, RN, the panel’s patient representative, called for any postmarketing studies that may be conducted by ReCor to include a good patient education program and also a way of documenting patient-reported outcomes.
“This would help us ascertain how happy people were if they were able to reduce their medication burden by, for example, one drug, or if they were willing to undergo a procedure to be able to stop taking so many antihypertensive medications,” she said.
The panel will meet again to review data on Medtronic’s Simplicity Spyral Renal Denervation System, which is also for patients with uncontrolled hypertension.
A version of this article appeared on Medscape.com.
in lowering blood pressure for adults with uncontrolled hypertension who may be inadequately responsive to, or who are intolerant of, antihypertensive medications.
The device is intended to be used in renal arteries with diameters of 3.0 to 8.0 mm.
After hearing data from three trials, RADIANCE-HTN SOLO, RADIANCE II, and RADIANCE-HTN TRIO, the 12-member panel unanimously agreed that there was “reasonable assurance” that the ReCor Paradise Ultrasound Renal Denervation System (ReCor Medical) was safe.
However, while most of the panel felt the device was effective, a few disagreed.
Keith Allen, MD, director of surgical research for the Mid-America Heart and Lung Surgeons, Kansas City, Mo., who was one of the three panel members who voted no regarding efficacy, stated that he had concerns about the duration and the degree of efficacy shown in the trials.
Mark Lockhart, MD, University of Alabama, Birmingham, also voted no. “I do think there was an effect for 2 months, but the duration of that positive effect appears to decline after that period of time.”
Benjamin Saville, PhD, echoed Dr. Lockhart’s concern: “The benefit is more short term, it is unclear what the long term benefit would be.”
Data from all three trials showed a significant drop in blood pressure with the device compared with pharmacologic therapy, but after 2 months of follow-up, that advantage disappeared.
The FDA highlighted data from the trials that showed that at 2 months, uRDN patients experienced significant reductions in systolic blood pressure compared with those treated with a sham device; however, by 6 months, there was a difference of only 1 mm Hg between the two groups.
“It seems when I look at 6 months and 12 months, the benefit is very tiny. We know the safety is fine, but a benefit of less than 1 mm Hg difference would not make me want to have an intervention,” said statistician Janet Wittes, PhD.
“I think the device is efficacious, even though there is not much difference between sham and treatment, but a big issue is the fact that half of our patients are not compliant. That will make the benefits over sham more clear,” noted Jim Blankenship, MD, professor of medicine and director of the division of cardiology at the University of New Mexico in Albuquerque.
John Hirshfeld Jr., MD, professor emeritus of medicine at the University of Pennsylvania, Philadelphia, said he voted yes on safety and efficacy but admitted he had some misgivings. “The sample size was small, but it is a novel tool to add to our tool box, and hopefully it will be used responsibly,” he said.
John Somberg, MD, professor emeritus of medicine, cardiology, and pharmacology, Rush University, Chicago, said the data on this procedure show “that antihypertensive medication works. Denervation is not superior to medications. It lowers blood pressure and is persistent, but when you can take the sham group to almost as good control as you get in the denervation group, that shows it can also be done with medicines.”
The panel wants to see results from additional studies in important subpopulations who are affected by hypertension, including Black people, women, the elderly, and people who already have cardiovascular risk factors, such as diabetes and heart failure.
Deneen Hesser, RN, the panel’s patient representative, called for any postmarketing studies that may be conducted by ReCor to include a good patient education program and also a way of documenting patient-reported outcomes.
“This would help us ascertain how happy people were if they were able to reduce their medication burden by, for example, one drug, or if they were willing to undergo a procedure to be able to stop taking so many antihypertensive medications,” she said.
The panel will meet again to review data on Medtronic’s Simplicity Spyral Renal Denervation System, which is also for patients with uncontrolled hypertension.
A version of this article appeared on Medscape.com.
Recall for Impella RP Flex labeling short on safety cautions
The current labeling doesn’t adequately describe safety precautions clinicians can take when patients’ clotting times are below the recommended range, according to the company and cited by the FDA.
The action applies to 65 units of the Impella RP Flex with SmartAssist, model number 1000323, first distributed by the company in November 2022, the statement notes.
Twelve related injuries but no deaths have been reported, the agency said.
The devices themselves are not part of the recall; clinicians may continue to use them, the FDA says.
However, “the use of affected catheters may cause serious adverse health consequences, including the risk of blood clots or particle deposits forming or death,” says the statement, which outlines instructions for mitigating the risk.
Abiomed says it is revising the label’s Instructions for Use section “to clarify the risk factors and recommendations related to the potential of thrombus formation or deposition.”
A version of this article first appeared on Medscape.com.
The current labeling doesn’t adequately describe safety precautions clinicians can take when patients’ clotting times are below the recommended range, according to the company and cited by the FDA.
The action applies to 65 units of the Impella RP Flex with SmartAssist, model number 1000323, first distributed by the company in November 2022, the statement notes.
Twelve related injuries but no deaths have been reported, the agency said.
The devices themselves are not part of the recall; clinicians may continue to use them, the FDA says.
However, “the use of affected catheters may cause serious adverse health consequences, including the risk of blood clots or particle deposits forming or death,” says the statement, which outlines instructions for mitigating the risk.
Abiomed says it is revising the label’s Instructions for Use section “to clarify the risk factors and recommendations related to the potential of thrombus formation or deposition.”
A version of this article first appeared on Medscape.com.
The current labeling doesn’t adequately describe safety precautions clinicians can take when patients’ clotting times are below the recommended range, according to the company and cited by the FDA.
The action applies to 65 units of the Impella RP Flex with SmartAssist, model number 1000323, first distributed by the company in November 2022, the statement notes.
Twelve related injuries but no deaths have been reported, the agency said.
The devices themselves are not part of the recall; clinicians may continue to use them, the FDA says.
However, “the use of affected catheters may cause serious adverse health consequences, including the risk of blood clots or particle deposits forming or death,” says the statement, which outlines instructions for mitigating the risk.
Abiomed says it is revising the label’s Instructions for Use section “to clarify the risk factors and recommendations related to the potential of thrombus formation or deposition.”
A version of this article first appeared on Medscape.com.
FDA warns AstraZeneca over ‘misleading claims’ about COPD drug
Promotional materials for the drug Breztri (budesonide/formoterol fumarate/glycopyrrolate inhaled) suggest that the drug has a positive effect on all-cause mortality for COPD patients, but the referenced clinical trial does not support that claim, the FDA letter states.
The FDA issued the warning letter on Aug. 4 and published the letter online on Aug. 15.
The sales aid highlights a 49% observed relative difference in time to all-cause mortality (ACM) over 1 year between Breztri and long-acting muscarinic antagonist/long-acting beta agonist (LAMA/LABA) inhalers.
Because of “statistical testing hierarchy failure” as well as confounding factors such as the removal of patients from inhaled corticosteroids (ICS) prior to entering the treatment arm of the trial, “no conclusions about the effect of Breztri on ACM can be drawn from the [clinical] trial,” the FDA wrote. “To date, no drug has been shown to improve ACM in COPD.”
The Breztri sales aid also states that there was a 20% reduction of severe exacerbations in patients using Breztri compared with patients using ICS/LABA. However, in the cited clinical trial, “the reduction in severe exacerbations was not statistically significant for patients treated with Breztri relative to comparator groups,” according to the FDA.
AstraZeneca has 15 working days from the receipt of the letter to respond in writing with “any plan for discontinuing use of such communications, or for ceasing distribution of Breztri,” the agency wrote.
A version of this article appeared on Medscape.com.
Promotional materials for the drug Breztri (budesonide/formoterol fumarate/glycopyrrolate inhaled) suggest that the drug has a positive effect on all-cause mortality for COPD patients, but the referenced clinical trial does not support that claim, the FDA letter states.
The FDA issued the warning letter on Aug. 4 and published the letter online on Aug. 15.
The sales aid highlights a 49% observed relative difference in time to all-cause mortality (ACM) over 1 year between Breztri and long-acting muscarinic antagonist/long-acting beta agonist (LAMA/LABA) inhalers.
Because of “statistical testing hierarchy failure” as well as confounding factors such as the removal of patients from inhaled corticosteroids (ICS) prior to entering the treatment arm of the trial, “no conclusions about the effect of Breztri on ACM can be drawn from the [clinical] trial,” the FDA wrote. “To date, no drug has been shown to improve ACM in COPD.”
The Breztri sales aid also states that there was a 20% reduction of severe exacerbations in patients using Breztri compared with patients using ICS/LABA. However, in the cited clinical trial, “the reduction in severe exacerbations was not statistically significant for patients treated with Breztri relative to comparator groups,” according to the FDA.
AstraZeneca has 15 working days from the receipt of the letter to respond in writing with “any plan for discontinuing use of such communications, or for ceasing distribution of Breztri,” the agency wrote.
A version of this article appeared on Medscape.com.
Promotional materials for the drug Breztri (budesonide/formoterol fumarate/glycopyrrolate inhaled) suggest that the drug has a positive effect on all-cause mortality for COPD patients, but the referenced clinical trial does not support that claim, the FDA letter states.
The FDA issued the warning letter on Aug. 4 and published the letter online on Aug. 15.
The sales aid highlights a 49% observed relative difference in time to all-cause mortality (ACM) over 1 year between Breztri and long-acting muscarinic antagonist/long-acting beta agonist (LAMA/LABA) inhalers.
Because of “statistical testing hierarchy failure” as well as confounding factors such as the removal of patients from inhaled corticosteroids (ICS) prior to entering the treatment arm of the trial, “no conclusions about the effect of Breztri on ACM can be drawn from the [clinical] trial,” the FDA wrote. “To date, no drug has been shown to improve ACM in COPD.”
The Breztri sales aid also states that there was a 20% reduction of severe exacerbations in patients using Breztri compared with patients using ICS/LABA. However, in the cited clinical trial, “the reduction in severe exacerbations was not statistically significant for patients treated with Breztri relative to comparator groups,” according to the FDA.
AstraZeneca has 15 working days from the receipt of the letter to respond in writing with “any plan for discontinuing use of such communications, or for ceasing distribution of Breztri,” the agency wrote.
A version of this article appeared on Medscape.com.
FDA okays first-ever new drug for rare bone disorder
Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.
“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.
Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.
It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.
The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.
Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.
According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”
The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.
Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”
The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”
Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”
Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).
A version of this article appeared on Medscape.com.
Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.
“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.
Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.
It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.
The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.
Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.
According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”
The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.
Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”
The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”
Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”
Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).
A version of this article appeared on Medscape.com.
Affecting roughly 400 people in the United States and 900 worldwide, FOP is an autosomal dominant condition in which bone develops in soft connective tissue areas of the body where it isn’t normally present (heterotopic ossification), such as the ligaments, tendons, and skeletal muscles. This leads to severe restriction in mobility and function, to the point that people lose the ability to feed or care for themselves. Most are completely disabled by age 30 years and median life expectancy is 56 years, with death often caused by bone formation around the rib cage restricting respiration.
“As a clinician caring for patients with FOP, I personally see the daily challenges and stresses that our patients and their families must contend with ... since the accumulation of heterotopic ossification in FOP is progressive, irreversible, and life altering. This medication is an important treatment option for our FOP community,” said endocrinologist Edward Hsiao, MD, professor of medicine at the University of California, San Francisco, in a statement from Ipsen.
Taken orally, palovarotene selectively targets the gamma subtype of retinoic acid receptors that regulate skeletal development and ectopic bone in the retinoid signaling pathway. The drug mediates interactions between these receptors, growth factors, and proteins within that pathway to reduce new abnormal bone formation.
It is now FDA approved for the treatment of FOP in female patients aged 8 years or older and male patients aged 10 years or older. The recommended dosing is 5 mg daily or weight-based equivalent for pediatric patients under 14 years of age, which can be modified or increased for flare-up symptoms. It is contraindicated during pregnancy.
The FDA approval was based on 18-month data from the phase 3, multicenter, open-label MOVE trial that included 107 adult and pediatric patients, over 10% of the world’s population with FOP. All received oral palovarotene and were compared with untreated individuals from a prior natural history study of the condition. The drug reduced annualized heterotopic ossification volume by 54%.
Side effects were typical of those seen with other systemic retinoid drugs, including mucocutaneous events such as dryness of the skin and mucous membranes, alopecia, drug eruption, rash, and pruritus, and musculoskeletal events, such as arthralgia and premature growth plate closure in growing children.
According to Dr. Hsiao, who was a MOVE investigator, the study “showed that Sohonos can decrease new heterotopic ossification, and that palovarotene can be tolerated by many patients with FOP. Sohonos is not for everyone. As with all medicines there are risks in this case especially for young children who may develop early growth plate closure. In addition, Sohonos has the same side effects as other retinoids.”
The FDA approval of palovarotene follows its rejection for marketing authorization in the European Union in July 2023.
Reached for comment, an Ipsen spokesperson said in an interview: “We reached the end of the regulatory process in the European Union for Sohonos and are disappointed the European Commission decided not to approved palovarotene for people with FOP in Europe.”
The company is developing another drug, fidrisertib, for treating FOP. A pivotal phase 2 trial for that drug is now recruiting patients. Asked where Ipsen might try to market fidrisertib, the spokesperson replied:“At this point, our focus is on the completion of the pivotal trial.”
Meanwhile, in the United States, the FOP community is celebrating the palovarotene approval. In a statement, Michelle Davis, executive director of the International Fibrodysplasia Ossificans Progressiva Association, said: “FOP is life altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close. ... The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”
Ipsen is offering a patient support program to assist with education, coverage, and reimbursement (1-866-435-5677).
A version of this article appeared on Medscape.com.