Hematology Times

Interior view of San Diego

Convention Center, site of

the 2016 ASH Annual Meeting

SAN DIEGO—Results of a phase 1 study suggest that SCD-101, a botanical extract based on an herbal medicine used in Nigeria to treat sickle cell disease (SCD), can reduce pain and fatigue in people with SCD.

The anti-sickling drug also improves the shape of red blood cells but doesn’t produce a change in hemoglobin, according to researchers.

Peter Gillette, MD, of SUNY Downstate in Brooklyn, New York, reported these results at the 2016 ASH Annual Meeting (abstract 121*).

A 6-month phase 2b study conducted previously in Nigeria showed that the herbal medicine Niprisan reduced pain crises and school absenteeism and raised hemoglobin levels compared to placebo.

Based on this study and positive preclinical activity, Dr Gillette and his colleagues undertook a phase 1 study to determine the safety of escalating doses of SCD-101.

Dr Gillette pointed out that Niprisan had been produced commercially in Nigeria but was later removed by the government from the commercial market because of production problems.

The researchers evaluated 23 patients with homozygous SCD or S/beta0 thalassemia.

Patients were aged 18 to 55 years with hemoglobin F of 15% or less and hemoglobin levels between 6.0 and 9.5 g/dL.

Patients could not have had hydroxyurea treatment within 6 months of enrollment, red blood cell transfusion within 3 months, or hospitalization within 4 weeks.

Patients received SCD-101 orally for 28 days administered 2 times daily (BID) or 3 times daily (TID). Doses were 550 mg BID, 1100 mg BID, 2200 mg BID, 4400 mg BID, and 2750 mg TID.

Dr Gillette explained that by distributing the highest dose 3 times over the course of a day, the researchers were able to decrease the side effects of bloating and flatulence on the highest dose.

“Interestingly, with the dose-distributed TID, we found that the hemoglobin had increased by 10%,” he said. “In other words, it appears that the effects are very short-acting, and that by going from a Q12 to a Q8 dosage, the hemoglobin suddenly looks like it might be significant, although this is not a significant change.”

Laboratory outcomes included hemoglobin and hemolysis (LDH, bilirubin, and reticulocyte measurements). Patient-reported outcomes included pain and fatigue.

The most common adverse events (AEs) were pain, flatulence, bloating, diarrhea, constipation, nausea, and headache.

Seven patients in the 2200 mg BID and 4400 mg BID cohorts had dose-related bloating, gas, flatulence or diarrhea, which subsided in a few days.

Patients in the 2750 mg TID cohort did not experience gas side effects.

The gastrointestinal symptoms were most likely dose-related from an excipient of SCD-101, Dr Gillette said.

He and his colleagues found no significant side effects after 28 days of dosing, and there were no dose reductions or interruptions due to drug-related AEs.

There were also no laboratory or electrocardiogram abnormalities.

“Almost all pain AEs stopped by day 13 in 22 of 23 patients,” Dr Gillette said.

“And unexpectedly, patients began to report that they slept better and had improved energy and cognition,” he noted.

Six patients in the 2200 and 4400 mg BID cohorts reported reduced fatigue as measured by the PROMIS fatigue questionnaire.

And 2 patients with ankle ulcers in the 2 highest dose cohorts reported improved healing.

Two weeks after treatment stopped, patients were almost back to baseline in terms of their chronic pain and fatigue levels, Dr Gillette said.

A parallel design, double-blind, placebo-controlled pilot study of the 2750 mg TID dose is ongoing.

Future studies include a crossover-design, exploratory study of the 2750 mg TID dose and a phase 2 parallel design study of the 2200 mg BID and 2750 mg TID doses.

While the researchers are uncertain about the mechanism of action of SCD-101, they hypothesize that its effects could be due to increased vascular flow, increased oxygen delivery, or a reduction in inflammation.

“This is a promising drug potentially for low-income countries or middle-income countries elsewhere in the world where gene therapy and transplant are really not that feasible,” Dr Gillette said.

Research for this study was supported in part by the National Heart, Lung, and Blood Institute and National Center for Complementary and Integrative Health of the National Institutes of Health.

*Information presented at the meeting differs from the abstract.

Interior view of San Diego

Convention Center, site of

the 2016 ASH Annual Meeting

SAN DIEGO—Results of a phase 1 study suggest that SCD-101, a botanical extract based on an herbal medicine used in Nigeria to treat sickle cell disease (SCD), can reduce pain and fatigue in people with SCD.

The anti-sickling drug also improves the shape of red blood cells but doesn’t produce a change in hemoglobin, according to researchers.

Peter Gillette, MD, of SUNY Downstate in Brooklyn, New York, reported these results at the 2016 ASH Annual Meeting (abstract 121*).

A 6-month phase 2b study conducted previously in Nigeria showed that the herbal medicine Niprisan reduced pain crises and school absenteeism and raised hemoglobin levels compared to placebo.

Based on this study and positive preclinical activity, Dr Gillette and his colleagues undertook a phase 1 study to determine the safety of escalating doses of SCD-101.

Dr Gillette pointed out that Niprisan had been produced commercially in Nigeria but was later removed by the government from the commercial market because of production problems.

The researchers evaluated 23 patients with homozygous SCD or S/beta0 thalassemia.

Patients were aged 18 to 55 years with hemoglobin F of 15% or less and hemoglobin levels between 6.0 and 9.5 g/dL.

Patients could not have had hydroxyurea treatment within 6 months of enrollment, red blood cell transfusion within 3 months, or hospitalization within 4 weeks.

Patients received SCD-101 orally for 28 days administered 2 times daily (BID) or 3 times daily (TID). Doses were 550 mg BID, 1100 mg BID, 2200 mg BID, 4400 mg BID, and 2750 mg TID.

Dr Gillette explained that by distributing the highest dose 3 times over the course of a day, the researchers were able to decrease the side effects of bloating and flatulence on the highest dose.

“Interestingly, with the dose-distributed TID, we found that the hemoglobin had increased by 10%,” he said. “In other words, it appears that the effects are very short-acting, and that by going from a Q12 to a Q8 dosage, the hemoglobin suddenly looks like it might be significant, although this is not a significant change.”

Laboratory outcomes included hemoglobin and hemolysis (LDH, bilirubin, and reticulocyte measurements). Patient-reported outcomes included pain and fatigue.

The most common adverse events (AEs) were pain, flatulence, bloating, diarrhea, constipation, nausea, and headache.

Seven patients in the 2200 mg BID and 4400 mg BID cohorts had dose-related bloating, gas, flatulence or diarrhea, which subsided in a few days.

Patients in the 2750 mg TID cohort did not experience gas side effects.

The gastrointestinal symptoms were most likely dose-related from an excipient of SCD-101, Dr Gillette said.

He and his colleagues found no significant side effects after 28 days of dosing, and there were no dose reductions or interruptions due to drug-related AEs.

There were also no laboratory or electrocardiogram abnormalities.

“Almost all pain AEs stopped by day 13 in 22 of 23 patients,” Dr Gillette said.

“And unexpectedly, patients began to report that they slept better and had improved energy and cognition,” he noted.

Six patients in the 2200 and 4400 mg BID cohorts reported reduced fatigue as measured by the PROMIS fatigue questionnaire.

And 2 patients with ankle ulcers in the 2 highest dose cohorts reported improved healing.

Two weeks after treatment stopped, patients were almost back to baseline in terms of their chronic pain and fatigue levels, Dr Gillette said.

A parallel design, double-blind, placebo-controlled pilot study of the 2750 mg TID dose is ongoing.

Future studies include a crossover-design, exploratory study of the 2750 mg TID dose and a phase 2 parallel design study of the 2200 mg BID and 2750 mg TID doses.

While the researchers are uncertain about the mechanism of action of SCD-101, they hypothesize that its effects could be due to increased vascular flow, increased oxygen delivery, or a reduction in inflammation.

“This is a promising drug potentially for low-income countries or middle-income countries elsewhere in the world where gene therapy and transplant are really not that feasible,” Dr Gillette said.

Research for this study was supported in part by the National Heart, Lung, and Blood Institute and National Center for Complementary and Integrative Health of the National Institutes of Health.

*Information presented at the meeting differs from the abstract.

Interior view of San Diego

Convention Center, site of

the 2016 ASH Annual Meeting

SAN DIEGO—Results of a phase 1 study suggest that SCD-101, a botanical extract based on an herbal medicine used in Nigeria to treat sickle cell disease (SCD), can reduce pain and fatigue in people with SCD.

The anti-sickling drug also improves the shape of red blood cells but doesn’t produce a change in hemoglobin, according to researchers.

Peter Gillette, MD, of SUNY Downstate in Brooklyn, New York, reported these results at the 2016 ASH Annual Meeting (abstract 121*).

A 6-month phase 2b study conducted previously in Nigeria showed that the herbal medicine Niprisan reduced pain crises and school absenteeism and raised hemoglobin levels compared to placebo.

Based on this study and positive preclinical activity, Dr Gillette and his colleagues undertook a phase 1 study to determine the safety of escalating doses of SCD-101.

Dr Gillette pointed out that Niprisan had been produced commercially in Nigeria but was later removed by the government from the commercial market because of production problems.

The researchers evaluated 23 patients with homozygous SCD or S/beta0 thalassemia.

Patients were aged 18 to 55 years with hemoglobin F of 15% or less and hemoglobin levels between 6.0 and 9.5 g/dL.

Patients could not have had hydroxyurea treatment within 6 months of enrollment, red blood cell transfusion within 3 months, or hospitalization within 4 weeks.

Patients received SCD-101 orally for 28 days administered 2 times daily (BID) or 3 times daily (TID). Doses were 550 mg BID, 1100 mg BID, 2200 mg BID, 4400 mg BID, and 2750 mg TID.

Dr Gillette explained that by distributing the highest dose 3 times over the course of a day, the researchers were able to decrease the side effects of bloating and flatulence on the highest dose.

“Interestingly, with the dose-distributed TID, we found that the hemoglobin had increased by 10%,” he said. “In other words, it appears that the effects are very short-acting, and that by going from a Q12 to a Q8 dosage, the hemoglobin suddenly looks like it might be significant, although this is not a significant change.”

Laboratory outcomes included hemoglobin and hemolysis (LDH, bilirubin, and reticulocyte measurements). Patient-reported outcomes included pain and fatigue.

The most common adverse events (AEs) were pain, flatulence, bloating, diarrhea, constipation, nausea, and headache.

Seven patients in the 2200 mg BID and 4400 mg BID cohorts had dose-related bloating, gas, flatulence or diarrhea, which subsided in a few days.

Patients in the 2750 mg TID cohort did not experience gas side effects.

The gastrointestinal symptoms were most likely dose-related from an excipient of SCD-101, Dr Gillette said.

He and his colleagues found no significant side effects after 28 days of dosing, and there were no dose reductions or interruptions due to drug-related AEs.

There were also no laboratory or electrocardiogram abnormalities.

“Almost all pain AEs stopped by day 13 in 22 of 23 patients,” Dr Gillette said.

“And unexpectedly, patients began to report that they slept better and had improved energy and cognition,” he noted.

Six patients in the 2200 and 4400 mg BID cohorts reported reduced fatigue as measured by the PROMIS fatigue questionnaire.

And 2 patients with ankle ulcers in the 2 highest dose cohorts reported improved healing.

Two weeks after treatment stopped, patients were almost back to baseline in terms of their chronic pain and fatigue levels, Dr Gillette said.

A parallel design, double-blind, placebo-controlled pilot study of the 2750 mg TID dose is ongoing.

Future studies include a crossover-design, exploratory study of the 2750 mg TID dose and a phase 2 parallel design study of the 2200 mg BID and 2750 mg TID doses.

While the researchers are uncertain about the mechanism of action of SCD-101, they hypothesize that its effects could be due to increased vascular flow, increased oxygen delivery, or a reduction in inflammation.

“This is a promising drug potentially for low-income countries or middle-income countries elsewhere in the world where gene therapy and transplant are really not that feasible,” Dr Gillette said.

Research for this study was supported in part by the National Heart, Lung, and Blood Institute and National Center for Complementary and Integrative Health of the National Institutes of Health.

*Information presented at the meeting differs from the abstract.

A small community in the Lake

Kariba area of southern Zambia

where malaria elimination

programs are underway.

Photo from Milen Nikolov

Malaria elimination in historically high transmission areas like southern Africa is possible with tools that are already available, according to new research.

The study suggests that high levels of vector control are key, and mass drug campaigns cannot make much of an impact without proper vector control.

Milen Nikolov, of the Institute for Disease Modeling in Bellevue, Washington, and colleagues reported these findings in PLOS Computational Biology.

The researchers said the Lake Kariba region of Southern Province, Zambia, is part of a multi-country malaria elimination effort. However, elimination in this area is challenging because villages with high and low malaria burden are interconnected through human travel.

With this in mind, the researchers combined a mathematical model of malaria transmission with field data from Zambia to test a variety of strategies for eliminating malaria in the Lake Kariba region.

The team used detailed spatial surveillance data from field studies—including household locations, climate, clinical malaria incidence, prevalence of malaria infections, and bednet usage rates—to construct a model of interconnected villages, then tested a variety of intervention scenarios to see which ones could lead to elimination.

The results indicate that elimination requires high, yet realistic, levels of vector control. And mass drug campaigns deployed to kill parasites in the human population can boost the chances of achieving elimination as long as vector control is well-implemented.

The researchers said this work suggests that elimination programs in sub-Saharan Africa should focus on how to achieve and maintain excellent coverage of vector control measures rather than spending resources on mass drug campaigns that are predicted to have little effect without well-implemented vector control already in place.

Human movement within the region should be targeted to achieve elimination, as should the importation of infections from outside the region. This is because both impact the likelihood of achieving elimination and understanding regional movement patterns can help guide strategies on targeting specific groups of at-risk people.

While no sub-Saharan African country has yet eliminated malaria, the researchers predict that regional malaria elimination is within reach with current tools, provided the efficacy and operational efficiency attained in southern Zambia can be extended and targeted to other key areas.

A small community in the Lake

Kariba area of southern Zambia

where malaria elimination

programs are underway.

Photo from Milen Nikolov

Malaria elimination in historically high transmission areas like southern Africa is possible with tools that are already available, according to new research.

The study suggests that high levels of vector control are key, and mass drug campaigns cannot make much of an impact without proper vector control.

Milen Nikolov, of the Institute for Disease Modeling in Bellevue, Washington, and colleagues reported these findings in PLOS Computational Biology.

The researchers said the Lake Kariba region of Southern Province, Zambia, is part of a multi-country malaria elimination effort. However, elimination in this area is challenging because villages with high and low malaria burden are interconnected through human travel.

With this in mind, the researchers combined a mathematical model of malaria transmission with field data from Zambia to test a variety of strategies for eliminating malaria in the Lake Kariba region.

The team used detailed spatial surveillance data from field studies—including household locations, climate, clinical malaria incidence, prevalence of malaria infections, and bednet usage rates—to construct a model of interconnected villages, then tested a variety of intervention scenarios to see which ones could lead to elimination.

The results indicate that elimination requires high, yet realistic, levels of vector control. And mass drug campaigns deployed to kill parasites in the human population can boost the chances of achieving elimination as long as vector control is well-implemented.

The researchers said this work suggests that elimination programs in sub-Saharan Africa should focus on how to achieve and maintain excellent coverage of vector control measures rather than spending resources on mass drug campaigns that are predicted to have little effect without well-implemented vector control already in place.

Human movement within the region should be targeted to achieve elimination, as should the importation of infections from outside the region. This is because both impact the likelihood of achieving elimination and understanding regional movement patterns can help guide strategies on targeting specific groups of at-risk people.

While no sub-Saharan African country has yet eliminated malaria, the researchers predict that regional malaria elimination is within reach with current tools, provided the efficacy and operational efficiency attained in southern Zambia can be extended and targeted to other key areas.

A small community in the Lake

Kariba area of southern Zambia

where malaria elimination

programs are underway.

Photo from Milen Nikolov

Malaria elimination in historically high transmission areas like southern Africa is possible with tools that are already available, according to new research.

The study suggests that high levels of vector control are key, and mass drug campaigns cannot make much of an impact without proper vector control.

Milen Nikolov, of the Institute for Disease Modeling in Bellevue, Washington, and colleagues reported these findings in PLOS Computational Biology.

The researchers said the Lake Kariba region of Southern Province, Zambia, is part of a multi-country malaria elimination effort. However, elimination in this area is challenging because villages with high and low malaria burden are interconnected through human travel.

With this in mind, the researchers combined a mathematical model of malaria transmission with field data from Zambia to test a variety of strategies for eliminating malaria in the Lake Kariba region.

The team used detailed spatial surveillance data from field studies—including household locations, climate, clinical malaria incidence, prevalence of malaria infections, and bednet usage rates—to construct a model of interconnected villages, then tested a variety of intervention scenarios to see which ones could lead to elimination.

The results indicate that elimination requires high, yet realistic, levels of vector control. And mass drug campaigns deployed to kill parasites in the human population can boost the chances of achieving elimination as long as vector control is well-implemented.

The researchers said this work suggests that elimination programs in sub-Saharan Africa should focus on how to achieve and maintain excellent coverage of vector control measures rather than spending resources on mass drug campaigns that are predicted to have little effect without well-implemented vector control already in place.

Human movement within the region should be targeted to achieve elimination, as should the importation of infections from outside the region. This is because both impact the likelihood of achieving elimination and understanding regional movement patterns can help guide strategies on targeting specific groups of at-risk people.

While no sub-Saharan African country has yet eliminated malaria, the researchers predict that regional malaria elimination is within reach with current tools, provided the efficacy and operational efficiency attained in southern Zambia can be extended and targeted to other key areas.

Pembrolizumab (Keytruda)

Photo courtesy of Merck

The US Food and Drug Administration (FDA) has granted priority review to the supplemental biologics license application (sBLA) for pembrolizumab (Keytruda®) as a treatment for patients with refractory classical Hodgkin lymphoma (cHL) and for cHL patients who have relapsed after 3 or more prior lines of therapy.

The sBLA will be reviewed under the FDA’s accelerated approval program. The target action date is March 15, 2017.

Pembrolizumab is a monoclonal antibody that binds to the PD-1 receptor and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the antitumor immune response.

The drug, which is being developed by Merck, already has FDA approval as a treatment for melanoma, lung cancer, and head and neck cancer.

Pembrolizumab also has breakthrough therapy designation as a treatment for relapsed/refractory cHL.

The current sBLA for pembrolizumab is seeking approval for the drug at a fixed dose of 200 mg, administered intravenously every 3 weeks.

This is the first application for regulatory approval of pembrolizumab in a hematologic malignancy.

The sBLA is supported by data from the phase 1 KEYNOTE-013 trial and the phase 2 KEYNOTE-087 trial.

Results from KEYNOTE-013 (in cHL patients) were presented at the 2014 ASH Annual Meeting, and results from KEYNOTE-087 were presented at the 2016 ASCO Annual Meeting.

Pembrolizumab (Keytruda)

Photo courtesy of Merck

The US Food and Drug Administration (FDA) has granted priority review to the supplemental biologics license application (sBLA) for pembrolizumab (Keytruda®) as a treatment for patients with refractory classical Hodgkin lymphoma (cHL) and for cHL patients who have relapsed after 3 or more prior lines of therapy.

The sBLA will be reviewed under the FDA’s accelerated approval program. The target action date is March 15, 2017.

Pembrolizumab is a monoclonal antibody that binds to the PD-1 receptor and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the antitumor immune response.

The drug, which is being developed by Merck, already has FDA approval as a treatment for melanoma, lung cancer, and head and neck cancer.

Pembrolizumab also has breakthrough therapy designation as a treatment for relapsed/refractory cHL.

The current sBLA for pembrolizumab is seeking approval for the drug at a fixed dose of 200 mg, administered intravenously every 3 weeks.

This is the first application for regulatory approval of pembrolizumab in a hematologic malignancy.

The sBLA is supported by data from the phase 1 KEYNOTE-013 trial and the phase 2 KEYNOTE-087 trial.

Results from KEYNOTE-013 (in cHL patients) were presented at the 2014 ASH Annual Meeting, and results from KEYNOTE-087 were presented at the 2016 ASCO Annual Meeting.

Pembrolizumab (Keytruda)

Photo courtesy of Merck

The US Food and Drug Administration (FDA) has granted priority review to the supplemental biologics license application (sBLA) for pembrolizumab (Keytruda®) as a treatment for patients with refractory classical Hodgkin lymphoma (cHL) and for cHL patients who have relapsed after 3 or more prior lines of therapy.

The sBLA will be reviewed under the FDA’s accelerated approval program. The target action date is March 15, 2017.

Pembrolizumab is a monoclonal antibody that binds to the PD-1 receptor and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the antitumor immune response.

The drug, which is being developed by Merck, already has FDA approval as a treatment for melanoma, lung cancer, and head and neck cancer.

Pembrolizumab also has breakthrough therapy designation as a treatment for relapsed/refractory cHL.

The current sBLA for pembrolizumab is seeking approval for the drug at a fixed dose of 200 mg, administered intravenously every 3 weeks.

This is the first application for regulatory approval of pembrolizumab in a hematologic malignancy.

The sBLA is supported by data from the phase 1 KEYNOTE-013 trial and the phase 2 KEYNOTE-087 trial.

Results from KEYNOTE-013 (in cHL patients) were presented at the 2014 ASH Annual Meeting, and results from KEYNOTE-087 were presented at the 2016 ASCO Annual Meeting.

Blood sample

Photo by Juan D. Alfonso

The US Food and Drug Administration (FDA) has issued an emergency use authorization (EUA) for Abbott Molecular Inc.’s RealTime ZIKA assay.

The EUA means the assay can be used by certified laboratories for the qualitative detection of RNA from Zika virus in human serum, EDTA plasma, and urine (collected alongside a patient-matched serum or plasma specimen).

Zika virus RNA is generally detectable in these specimens during the acute phase of infection.

According to updated guidance from the US Centers for Disease Control and Prevention (CDC), Zika virus RNA is detectable up to 14 days in serum and urine (possibly longer in urine), following the onset of symptoms, if present. Positive results are indicative of current infection.

The FDA’s decision to grant an EUA means Abbott’s RealTime ZIKA assay can be used in individuals who meet CDC Zika virus clinical criteria (eg, clinical signs and symptoms associated with Zika virus infection) and/or CDC Zika virus epidemiological criteria (eg, history of residence in or travel to a geographic region with active Zika transmission at the time of travel, or other epidemiological criteria for which Zika virus testing may be indicated).

The assay can be used by laboratories in the US that are certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), 42 U.S.C. §263a, to perform high complexity tests, or by similarly qualified non-US laboratories, pursuant to section 564 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. § 360bbb-3).

The EUA does not mean Abbott’s RealTime ZIKA assay is FDA cleared or approved.

An EUA allows for the use of unapproved medical products or unapproved uses of approved medical products in an emergency.

The products must be used to diagnose, treat, or prevent serious or life-threatening conditions caused by chemical, biological, radiological, or nuclear threat agents, when there are no adequate alternatives.

This means Abbott’s RealTime ZIKA assay is only authorized as long as circumstances exist to justify the emergency use of in vitro diagnostics for the detection of Zika virus, unless the authorization is terminated or revoked sooner.

Blood sample

Photo by Juan D. Alfonso

The US Food and Drug Administration (FDA) has issued an emergency use authorization (EUA) for Abbott Molecular Inc.’s RealTime ZIKA assay.

The EUA means the assay can be used by certified laboratories for the qualitative detection of RNA from Zika virus in human serum, EDTA plasma, and urine (collected alongside a patient-matched serum or plasma specimen).

Zika virus RNA is generally detectable in these specimens during the acute phase of infection.

According to updated guidance from the US Centers for Disease Control and Prevention (CDC), Zika virus RNA is detectable up to 14 days in serum and urine (possibly longer in urine), following the onset of symptoms, if present. Positive results are indicative of current infection.

The FDA’s decision to grant an EUA means Abbott’s RealTime ZIKA assay can be used in individuals who meet CDC Zika virus clinical criteria (eg, clinical signs and symptoms associated with Zika virus infection) and/or CDC Zika virus epidemiological criteria (eg, history of residence in or travel to a geographic region with active Zika transmission at the time of travel, or other epidemiological criteria for which Zika virus testing may be indicated).

The assay can be used by laboratories in the US that are certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), 42 U.S.C. §263a, to perform high complexity tests, or by similarly qualified non-US laboratories, pursuant to section 564 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. § 360bbb-3).

The EUA does not mean Abbott’s RealTime ZIKA assay is FDA cleared or approved.

An EUA allows for the use of unapproved medical products or unapproved uses of approved medical products in an emergency.

The products must be used to diagnose, treat, or prevent serious or life-threatening conditions caused by chemical, biological, radiological, or nuclear threat agents, when there are no adequate alternatives.

This means Abbott’s RealTime ZIKA assay is only authorized as long as circumstances exist to justify the emergency use of in vitro diagnostics for the detection of Zika virus, unless the authorization is terminated or revoked sooner.

Blood sample

Photo by Juan D. Alfonso

The US Food and Drug Administration (FDA) has issued an emergency use authorization (EUA) for Abbott Molecular Inc.’s RealTime ZIKA assay.

The EUA means the assay can be used by certified laboratories for the qualitative detection of RNA from Zika virus in human serum, EDTA plasma, and urine (collected alongside a patient-matched serum or plasma specimen).

Zika virus RNA is generally detectable in these specimens during the acute phase of infection.

According to updated guidance from the US Centers for Disease Control and Prevention (CDC), Zika virus RNA is detectable up to 14 days in serum and urine (possibly longer in urine), following the onset of symptoms, if present. Positive results are indicative of current infection.

The FDA’s decision to grant an EUA means Abbott’s RealTime ZIKA assay can be used in individuals who meet CDC Zika virus clinical criteria (eg, clinical signs and symptoms associated with Zika virus infection) and/or CDC Zika virus epidemiological criteria (eg, history of residence in or travel to a geographic region with active Zika transmission at the time of travel, or other epidemiological criteria for which Zika virus testing may be indicated).

The assay can be used by laboratories in the US that are certified under the Clinical Laboratory Improvement Amendments of 1988 (CLIA), 42 U.S.C. §263a, to perform high complexity tests, or by similarly qualified non-US laboratories, pursuant to section 564 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. § 360bbb-3).

The EUA does not mean Abbott’s RealTime ZIKA assay is FDA cleared or approved.

An EUA allows for the use of unapproved medical products or unapproved uses of approved medical products in an emergency.

The products must be used to diagnose, treat, or prevent serious or life-threatening conditions caused by chemical, biological, radiological, or nuclear threat agents, when there are no adequate alternatives.

This means Abbott’s RealTime ZIKA assay is only authorized as long as circumstances exist to justify the emergency use of in vitro diagnostics for the detection of Zika virus, unless the authorization is terminated or revoked sooner.

Lab mice

Photo by Aaron Logan

Researchers have created a patch designed to monitor a patient’s blood and release heparin as needed to prevent thrombosis.

In mice, the patch successfully released heparin into the bloodstream and proved more effective at preventing thrombosis than an injection of heparin.

Zhen Gu, PhD, of the University of North Carolina at Chapel Hill, and his colleagues developed the patch and described it in Advanced Materials.

“Our goal was to generate a patch that can monitor a patient’s blood and release additional drugs when necessary; effectively, a self-regulating system,” Dr Gu said.

The patch incorporates microneedles made of a polymer that consists of hyaluronic acid (HA) and the drug heparin. The polymer has been modified to be responsive to thrombin.

When elevated levels of thrombin enzymes in the bloodstream come into contact with the microneedle, the enzymes break the amino acid chains that bind the heparin to the HA, releasing the heparin into the bloodstream.

“The more thrombin there is in the bloodstream, the more heparin is needed to reduce clotting,” said study author Yuqi Zhang, a PhD student in Dr Gu’s lab. “So we created a disposable patch in which the more thrombin there is in the bloodstream, the more heparin is released.”

“We will further enhance the loading amount of drug in the patch,” said study author Jicheng Yu, a PhD student in Dr Gu’s lab.

“The amount of heparin in a patch can be tailored to a patient’s specific needs and replaced daily, or less often, as needed. But the amount of heparin being released into the patient at any given moment will be determined by the thrombin levels in the patient’s blood.”

Experiments in mice

The researchers tested the patch in a mouse model of thrombosis. The mice were injected with large doses of thrombin (1000 U kg^-1) to induce an acute thromboembolism, which can lead to death in about 92% of mice.

The mice were then randomized into 5 different treatment groups:

• Intravenous heparin injection
• Empty HA microneedle patch
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch
• Non-responsive HA-heparin microneedle patch (heparin dose: 200 U kg⁻¹).

The mice that received heparin via injection were treated before thrombosis induction. The microneedle patches were applied to the dorsum skin of the mice 10 minutes before the challenge.

All of the mice with the HA microneedle patch or the non-responsive HA-heparin microneedle patch died within 15 minutes of the thrombin injection.

All of the mice that received the heparin injection, heparin microneedle patch, or the thrombin-responsive HA-heparin microneedle patch survived the 15 minutes.

In a second experiment, mice received a thrombin injection 6 hours after treatment, and treatment consisted of:

• Heparin injection
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch.

Fifteen minutes after the thrombin injection, death had occurred in 80% or more of the mice that received the heparin injection and the mice treated with the heparin microneedle patch.

But all of the mice treated with the thrombin-responsive HA-heparin microneedle patch survived.

The researchers also noted that staining of lung sections revealed the “superior anticoagulant capacity” of the thrombin-responsive HA-heparin microneedle patch.

The team observed “insignificant differences” in the lungs of healthy mice and mice treated with the thrombin-responsive HA-heparin microneedle patch.

However, mice that received a heparin injection or treatment with the heparin microneedle patch had intravascular and interstitial hemorrhage, blocked blood vessels, and atelectasis.

“We’re excited about the possibility of using a closed-loop, self-regulating smart patch to help treat a condition that affects thousands of people every year, while hopefully also driving down treatment costs,” Dr Gu said. “This paper represents a good first step, and we’re now looking for funding to perform additional preclinical testing.”

Lab mice

Photo by Aaron Logan

Researchers have created a patch designed to monitor a patient’s blood and release heparin as needed to prevent thrombosis.

In mice, the patch successfully released heparin into the bloodstream and proved more effective at preventing thrombosis than an injection of heparin.

Zhen Gu, PhD, of the University of North Carolina at Chapel Hill, and his colleagues developed the patch and described it in Advanced Materials.

“Our goal was to generate a patch that can monitor a patient’s blood and release additional drugs when necessary; effectively, a self-regulating system,” Dr Gu said.

The patch incorporates microneedles made of a polymer that consists of hyaluronic acid (HA) and the drug heparin. The polymer has been modified to be responsive to thrombin.

When elevated levels of thrombin enzymes in the bloodstream come into contact with the microneedle, the enzymes break the amino acid chains that bind the heparin to the HA, releasing the heparin into the bloodstream.

“The more thrombin there is in the bloodstream, the more heparin is needed to reduce clotting,” said study author Yuqi Zhang, a PhD student in Dr Gu’s lab. “So we created a disposable patch in which the more thrombin there is in the bloodstream, the more heparin is released.”

“We will further enhance the loading amount of drug in the patch,” said study author Jicheng Yu, a PhD student in Dr Gu’s lab.

“The amount of heparin in a patch can be tailored to a patient’s specific needs and replaced daily, or less often, as needed. But the amount of heparin being released into the patient at any given moment will be determined by the thrombin levels in the patient’s blood.”

Experiments in mice

The researchers tested the patch in a mouse model of thrombosis. The mice were injected with large doses of thrombin (1000 U kg^-1) to induce an acute thromboembolism, which can lead to death in about 92% of mice.

The mice were then randomized into 5 different treatment groups:

• Intravenous heparin injection
• Empty HA microneedle patch
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch
• Non-responsive HA-heparin microneedle patch (heparin dose: 200 U kg⁻¹).

The mice that received heparin via injection were treated before thrombosis induction. The microneedle patches were applied to the dorsum skin of the mice 10 minutes before the challenge.

All of the mice with the HA microneedle patch or the non-responsive HA-heparin microneedle patch died within 15 minutes of the thrombin injection.

All of the mice that received the heparin injection, heparin microneedle patch, or the thrombin-responsive HA-heparin microneedle patch survived the 15 minutes.

In a second experiment, mice received a thrombin injection 6 hours after treatment, and treatment consisted of:

• Heparin injection
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch.

Fifteen minutes after the thrombin injection, death had occurred in 80% or more of the mice that received the heparin injection and the mice treated with the heparin microneedle patch.

But all of the mice treated with the thrombin-responsive HA-heparin microneedle patch survived.

The researchers also noted that staining of lung sections revealed the “superior anticoagulant capacity” of the thrombin-responsive HA-heparin microneedle patch.

The team observed “insignificant differences” in the lungs of healthy mice and mice treated with the thrombin-responsive HA-heparin microneedle patch.

However, mice that received a heparin injection or treatment with the heparin microneedle patch had intravascular and interstitial hemorrhage, blocked blood vessels, and atelectasis.

“We’re excited about the possibility of using a closed-loop, self-regulating smart patch to help treat a condition that affects thousands of people every year, while hopefully also driving down treatment costs,” Dr Gu said. “This paper represents a good first step, and we’re now looking for funding to perform additional preclinical testing.”

Lab mice

Photo by Aaron Logan

Researchers have created a patch designed to monitor a patient’s blood and release heparin as needed to prevent thrombosis.

In mice, the patch successfully released heparin into the bloodstream and proved more effective at preventing thrombosis than an injection of heparin.

Zhen Gu, PhD, of the University of North Carolina at Chapel Hill, and his colleagues developed the patch and described it in Advanced Materials.

“Our goal was to generate a patch that can monitor a patient’s blood and release additional drugs when necessary; effectively, a self-regulating system,” Dr Gu said.

The patch incorporates microneedles made of a polymer that consists of hyaluronic acid (HA) and the drug heparin. The polymer has been modified to be responsive to thrombin.

When elevated levels of thrombin enzymes in the bloodstream come into contact with the microneedle, the enzymes break the amino acid chains that bind the heparin to the HA, releasing the heparin into the bloodstream.

“The more thrombin there is in the bloodstream, the more heparin is needed to reduce clotting,” said study author Yuqi Zhang, a PhD student in Dr Gu’s lab. “So we created a disposable patch in which the more thrombin there is in the bloodstream, the more heparin is released.”

“We will further enhance the loading amount of drug in the patch,” said study author Jicheng Yu, a PhD student in Dr Gu’s lab.

“The amount of heparin in a patch can be tailored to a patient’s specific needs and replaced daily, or less often, as needed. But the amount of heparin being released into the patient at any given moment will be determined by the thrombin levels in the patient’s blood.”

Experiments in mice

The researchers tested the patch in a mouse model of thrombosis. The mice were injected with large doses of thrombin (1000 U kg^-1) to induce an acute thromboembolism, which can lead to death in about 92% of mice.

The mice were then randomized into 5 different treatment groups:

• Intravenous heparin injection
• Empty HA microneedle patch
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch
• Non-responsive HA-heparin microneedle patch (heparin dose: 200 U kg⁻¹).

The mice that received heparin via injection were treated before thrombosis induction. The microneedle patches were applied to the dorsum skin of the mice 10 minutes before the challenge.

All of the mice with the HA microneedle patch or the non-responsive HA-heparin microneedle patch died within 15 minutes of the thrombin injection.

All of the mice that received the heparin injection, heparin microneedle patch, or the thrombin-responsive HA-heparin microneedle patch survived the 15 minutes.

In a second experiment, mice received a thrombin injection 6 hours after treatment, and treatment consisted of:

• Heparin injection
• HA microneedle patch encapsulating free heparin
• Thrombin-responsive HA-heparin microneedle patch.

Fifteen minutes after the thrombin injection, death had occurred in 80% or more of the mice that received the heparin injection and the mice treated with the heparin microneedle patch.

But all of the mice treated with the thrombin-responsive HA-heparin microneedle patch survived.

The researchers also noted that staining of lung sections revealed the “superior anticoagulant capacity” of the thrombin-responsive HA-heparin microneedle patch.

The team observed “insignificant differences” in the lungs of healthy mice and mice treated with the thrombin-responsive HA-heparin microneedle patch.

However, mice that received a heparin injection or treatment with the heparin microneedle patch had intravascular and interstitial hemorrhage, blocked blood vessels, and atelectasis.

“We’re excited about the possibility of using a closed-loop, self-regulating smart patch to help treat a condition that affects thousands of people every year, while hopefully also driving down treatment costs,” Dr Gu said. “This paper represents a good first step, and we’re now looking for funding to perform additional preclinical testing.”

NCCN Guidelines for Patients®:

Nausea and Vomiting

©NCCN® 2016

The National Comprehensive Cancer Network (NCCN) has released new educational materials designed to help cancer patients combat nausea and vomiting.

The NCCN Guidelines for Patients® for Nausea and Vomiting and NCCN Quick Guide™ for Nausea and Vomiting are the first patient resources from NCCN to focus specifically on supportive care.

The resources are available on NCCN.org/patients and via the NCCN Patient Guides for Cancer mobile app.

NCCN Guidelines for Patients are patient-friendly translations of the NCCN Clinical Practice Guidelines in Oncology. Each resource features guidance from US cancer centers designed to help people living with cancer talk with their physicians about the best treatment options for their disease.

NCCN Quick Guide™ sheets are 1-page summaries of key points in the patient guidelines. They include elements such as “questions to ask your doctor,” a glossary of terms, and medical illustrations of anatomy, tests, and treatments.

The NCCN Guidelines for Patients for Nausea and Vomiting:

• Explain how these side effects are related to cancer treatment
• List cancer treatments that can cause nausea and vomiting
• Detail methods of preventing and treating these side effects
• Outline methods of coping with nausea and vomiting
• Provide a list of resources for information and support.

“At NCCN, our mission is to improve the lives of patients with cancer, and we are excited to be able to provide the information that will help patients better understand this common side effect of cancer treatment,” said Marcie R. Reeder, executive director of the NCCN Foundation.

“The NCCN Guidelines for Patients for Nausea and Vomiting are the first of a highly anticipated library of supportive care resources that provide patients with the same information their doctors use.”

NCCN Guidelines for Patients®:

Nausea and Vomiting

©NCCN® 2016

The National Comprehensive Cancer Network (NCCN) has released new educational materials designed to help cancer patients combat nausea and vomiting.

The NCCN Guidelines for Patients® for Nausea and Vomiting and NCCN Quick Guide™ for Nausea and Vomiting are the first patient resources from NCCN to focus specifically on supportive care.

The resources are available on NCCN.org/patients and via the NCCN Patient Guides for Cancer mobile app.

NCCN Guidelines for Patients are patient-friendly translations of the NCCN Clinical Practice Guidelines in Oncology. Each resource features guidance from US cancer centers designed to help people living with cancer talk with their physicians about the best treatment options for their disease.

NCCN Quick Guide™ sheets are 1-page summaries of key points in the patient guidelines. They include elements such as “questions to ask your doctor,” a glossary of terms, and medical illustrations of anatomy, tests, and treatments.

The NCCN Guidelines for Patients for Nausea and Vomiting:

• Explain how these side effects are related to cancer treatment
• List cancer treatments that can cause nausea and vomiting
• Detail methods of preventing and treating these side effects
• Outline methods of coping with nausea and vomiting
• Provide a list of resources for information and support.

“At NCCN, our mission is to improve the lives of patients with cancer, and we are excited to be able to provide the information that will help patients better understand this common side effect of cancer treatment,” said Marcie R. Reeder, executive director of the NCCN Foundation.

“The NCCN Guidelines for Patients for Nausea and Vomiting are the first of a highly anticipated library of supportive care resources that provide patients with the same information their doctors use.”

NCCN Guidelines for Patients®:

Nausea and Vomiting

©NCCN® 2016

The National Comprehensive Cancer Network (NCCN) has released new educational materials designed to help cancer patients combat nausea and vomiting.

The NCCN Guidelines for Patients® for Nausea and Vomiting and NCCN Quick Guide™ for Nausea and Vomiting are the first patient resources from NCCN to focus specifically on supportive care.

The resources are available on NCCN.org/patients and via the NCCN Patient Guides for Cancer mobile app.

NCCN Guidelines for Patients are patient-friendly translations of the NCCN Clinical Practice Guidelines in Oncology. Each resource features guidance from US cancer centers designed to help people living with cancer talk with their physicians about the best treatment options for their disease.

NCCN Quick Guide™ sheets are 1-page summaries of key points in the patient guidelines. They include elements such as “questions to ask your doctor,” a glossary of terms, and medical illustrations of anatomy, tests, and treatments.

The NCCN Guidelines for Patients for Nausea and Vomiting:

• Explain how these side effects are related to cancer treatment
• List cancer treatments that can cause nausea and vomiting
• Detail methods of preventing and treating these side effects
• Outline methods of coping with nausea and vomiting
• Provide a list of resources for information and support.

“At NCCN, our mission is to improve the lives of patients with cancer, and we are excited to be able to provide the information that will help patients better understand this common side effect of cancer treatment,” said Marcie R. Reeder, executive director of the NCCN Foundation.

“The NCCN Guidelines for Patients for Nausea and Vomiting are the first of a highly anticipated library of supportive care resources that provide patients with the same information their doctors use.”

Testosterone cypionate

(Depo-Testosterone)

Starting testosterone treatment is associated with an increased risk of venous thromboembolism (VTE) that peaks within 6 months and declines thereafter, according to research published in The BMJ.

Previous studies have reported contradictory results regarding testosterone use and VTE.

Researchers involved in the current study believe that failure to investigate the timing and duration of testosterone use may explain the conflicting findings.

For this study, David Handelsman, MBBS, PhD, of the University of Sydney in New South Wales, Australia, and his colleagues set out to determine the risk of VTE associated with use of testosterone treatment in men, focusing particularly on the timing of the risk.

The study involved data from 19,215 patients with confirmed VTE and 909,530 age-matched controls registered with the UK Clinical Practice Research Database between January 2001 and May 2013.

The researchers divided subjects into 3 mutually exclusive testosterone exposure groups: current treatment, recent treatment, and no treatment in the previous 2 years. The “current treatment” group was subdivided into durations of more or less than 6 months.

After adjusting for confounding factors, the researchers estimated rates of VTE.

The adjusted rate ratio of VTE was 1.25 for current testosterone treatment as compared to no testosterone treatment.

In the first 6 months of treatment, the rate ratio of VTE was 1.63, which corresponded to 10 additional cases of VTE above the base rate of 15.8 per 10,000 person-years.

The risk of VTE declined after more than 6 months of treatment and after treatment stopped. The rate ratio after more than 6 months of treatment was 1.00. After treatment stopped, the rate ratio was 0.68.

The researchers noted that this is an observational study, so no firm conclusions can be drawn about cause and effect. The team also stressed that the increased risks observed are temporary and still relatively low in absolute terms.

Nevertheless, they said their study suggests “a transient increase in the risk of venous thromboembolism that peaks during the first 3 to 6 months and declines gradually thereafter.”

The team said additional research is needed to confirm the temporal increase in the risk of VTE they observed as well as the absence of risk with long-term testosterone use.

Testosterone cypionate

(Depo-Testosterone)

Starting testosterone treatment is associated with an increased risk of venous thromboembolism (VTE) that peaks within 6 months and declines thereafter, according to research published in The BMJ.

Previous studies have reported contradictory results regarding testosterone use and VTE.

Researchers involved in the current study believe that failure to investigate the timing and duration of testosterone use may explain the conflicting findings.

For this study, David Handelsman, MBBS, PhD, of the University of Sydney in New South Wales, Australia, and his colleagues set out to determine the risk of VTE associated with use of testosterone treatment in men, focusing particularly on the timing of the risk.

The study involved data from 19,215 patients with confirmed VTE and 909,530 age-matched controls registered with the UK Clinical Practice Research Database between January 2001 and May 2013.

The researchers divided subjects into 3 mutually exclusive testosterone exposure groups: current treatment, recent treatment, and no treatment in the previous 2 years. The “current treatment” group was subdivided into durations of more or less than 6 months.

After adjusting for confounding factors, the researchers estimated rates of VTE.

The adjusted rate ratio of VTE was 1.25 for current testosterone treatment as compared to no testosterone treatment.

In the first 6 months of treatment, the rate ratio of VTE was 1.63, which corresponded to 10 additional cases of VTE above the base rate of 15.8 per 10,000 person-years.

The risk of VTE declined after more than 6 months of treatment and after treatment stopped. The rate ratio after more than 6 months of treatment was 1.00. After treatment stopped, the rate ratio was 0.68.

The researchers noted that this is an observational study, so no firm conclusions can be drawn about cause and effect. The team also stressed that the increased risks observed are temporary and still relatively low in absolute terms.

Nevertheless, they said their study suggests “a transient increase in the risk of venous thromboembolism that peaks during the first 3 to 6 months and declines gradually thereafter.”

The team said additional research is needed to confirm the temporal increase in the risk of VTE they observed as well as the absence of risk with long-term testosterone use.

Testosterone cypionate

(Depo-Testosterone)

Starting testosterone treatment is associated with an increased risk of venous thromboembolism (VTE) that peaks within 6 months and declines thereafter, according to research published in The BMJ.

Previous studies have reported contradictory results regarding testosterone use and VTE.

Researchers involved in the current study believe that failure to investigate the timing and duration of testosterone use may explain the conflicting findings.

For this study, David Handelsman, MBBS, PhD, of the University of Sydney in New South Wales, Australia, and his colleagues set out to determine the risk of VTE associated with use of testosterone treatment in men, focusing particularly on the timing of the risk.

The study involved data from 19,215 patients with confirmed VTE and 909,530 age-matched controls registered with the UK Clinical Practice Research Database between January 2001 and May 2013.

The researchers divided subjects into 3 mutually exclusive testosterone exposure groups: current treatment, recent treatment, and no treatment in the previous 2 years. The “current treatment” group was subdivided into durations of more or less than 6 months.

After adjusting for confounding factors, the researchers estimated rates of VTE.

The adjusted rate ratio of VTE was 1.25 for current testosterone treatment as compared to no testosterone treatment.

In the first 6 months of treatment, the rate ratio of VTE was 1.63, which corresponded to 10 additional cases of VTE above the base rate of 15.8 per 10,000 person-years.

The risk of VTE declined after more than 6 months of treatment and after treatment stopped. The rate ratio after more than 6 months of treatment was 1.00. After treatment stopped, the rate ratio was 0.68.

The researchers noted that this is an observational study, so no firm conclusions can be drawn about cause and effect. The team also stressed that the increased risks observed are temporary and still relatively low in absolute terms.

Nevertheless, they said their study suggests “a transient increase in the risk of venous thromboembolism that peaks during the first 3 to 6 months and declines gradually thereafter.”

The team said additional research is needed to confirm the temporal increase in the risk of VTE they observed as well as the absence of risk with long-term testosterone use.

A sickled red blood cell

beside a normal one

Image by Betty Pace

The European Commission (EC) has designated GBT440 as an orphan medicinal product for the treatment of sickle cell disease (SCD).

GBT440 is being developed as a potentially disease-modifying therapy for SCD. The drug works by increasing hemoglobin’s affinity for oxygen.

Since oxygenated sickle hemoglobin does not polymerize, it is believed that GBT440 blocks polymerization and the resultant sickling of red blood cells.

If GBT440 can restore normal hemoglobin function and improve oxygen delivery, the drug may be capable of modifying the progression of SCD.

Preclinical research published in the British Journal of Haematology earlier this year suggests that GBT440 is disease-modifying.

Early results from an ongoing phase 1/2 study of GBT440, which were presented at the 2015 ASH Annual Meeting, appeared promising as well.

Results from that study suggest that GBT440 can increase hemoglobin levels while decreasing reticulocyte counts, erythropoietin levels, and sickle cell counts.

Researchers also found the drug to be well tolerated, with no serious adverse events attributed to GBT440.

“Receiving orphan designation from the EC marks a significant milestone both for the SCD community and for GBT [Global Blood Therapeutics Inc.],” said Ted W. Love, MD, president and chief executive officer of Global Blood Therapeutics Inc., the company developing GBT440.

“SCD is a devastatingly severe disease with limited treatment options, and this designation, together with our fast track and orphan drug designations by the United States Food and Drug Administration, reflect the recognition of the broader regulatory community of this urgent unmet medical need.”

The EC grants orphan designation to therapies intended to treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides the company developing a drug with regulatory and financial incentives, including protocol assistance, 10 years of market exclusivity once the drug is approved, and reductions in, or exemptions from, fees.

A sickled red blood cell

beside a normal one

Image by Betty Pace

The European Commission (EC) has designated GBT440 as an orphan medicinal product for the treatment of sickle cell disease (SCD).

GBT440 is being developed as a potentially disease-modifying therapy for SCD. The drug works by increasing hemoglobin’s affinity for oxygen.

Since oxygenated sickle hemoglobin does not polymerize, it is believed that GBT440 blocks polymerization and the resultant sickling of red blood cells.

If GBT440 can restore normal hemoglobin function and improve oxygen delivery, the drug may be capable of modifying the progression of SCD.

Preclinical research published in the British Journal of Haematology earlier this year suggests that GBT440 is disease-modifying.

Early results from an ongoing phase 1/2 study of GBT440, which were presented at the 2015 ASH Annual Meeting, appeared promising as well.

Results from that study suggest that GBT440 can increase hemoglobin levels while decreasing reticulocyte counts, erythropoietin levels, and sickle cell counts.

Researchers also found the drug to be well tolerated, with no serious adverse events attributed to GBT440.

“Receiving orphan designation from the EC marks a significant milestone both for the SCD community and for GBT [Global Blood Therapeutics Inc.],” said Ted W. Love, MD, president and chief executive officer of Global Blood Therapeutics Inc., the company developing GBT440.

“SCD is a devastatingly severe disease with limited treatment options, and this designation, together with our fast track and orphan drug designations by the United States Food and Drug Administration, reflect the recognition of the broader regulatory community of this urgent unmet medical need.”

The EC grants orphan designation to therapies intended to treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides the company developing a drug with regulatory and financial incentives, including protocol assistance, 10 years of market exclusivity once the drug is approved, and reductions in, or exemptions from, fees.

A sickled red blood cell

beside a normal one

Image by Betty Pace

The European Commission (EC) has designated GBT440 as an orphan medicinal product for the treatment of sickle cell disease (SCD).

GBT440 is being developed as a potentially disease-modifying therapy for SCD. The drug works by increasing hemoglobin’s affinity for oxygen.

Since oxygenated sickle hemoglobin does not polymerize, it is believed that GBT440 blocks polymerization and the resultant sickling of red blood cells.

If GBT440 can restore normal hemoglobin function and improve oxygen delivery, the drug may be capable of modifying the progression of SCD.

Preclinical research published in the British Journal of Haematology earlier this year suggests that GBT440 is disease-modifying.

Early results from an ongoing phase 1/2 study of GBT440, which were presented at the 2015 ASH Annual Meeting, appeared promising as well.

Results from that study suggest that GBT440 can increase hemoglobin levels while decreasing reticulocyte counts, erythropoietin levels, and sickle cell counts.

Researchers also found the drug to be well tolerated, with no serious adverse events attributed to GBT440.

“Receiving orphan designation from the EC marks a significant milestone both for the SCD community and for GBT [Global Blood Therapeutics Inc.],” said Ted W. Love, MD, president and chief executive officer of Global Blood Therapeutics Inc., the company developing GBT440.

“SCD is a devastatingly severe disease with limited treatment options, and this designation, together with our fast track and orphan drug designations by the United States Food and Drug Administration, reflect the recognition of the broader regulatory community of this urgent unmet medical need.”

The EC grants orphan designation to therapies intended to treat life-threatening or chronically debilitating conditions affecting no more than 5 in 10,000 people in the European Union, and where no satisfactory treatment is available.

Orphan designation provides the company developing a drug with regulatory and financial incentives, including protocol assistance, 10 years of market exclusivity once the drug is approved, and reductions in, or exemptions from, fees.

Pomalidomide (Pomalyst)

Photo from Business Wire

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that pomalidomide be made available through the National Health Service (NHS).

NICE is recommending pomalidomide be available for use in combination with low-dose dexamethasone to treat adults with multiple myeloma who have received at least 3 previous treatments, including lenalidomide and bortezomib.

NICE previously evaluated pomalidomide in 2015 and said it could not recommend the drug, as analyses suggested pomalidomide doesn’t provide enough benefit to justify its high price.

Since that time, a committee advising NICE has reviewed additional data on pomalidomide.

And Celgene, the company that makes pomalidomide, has agreed to provide the NHS with a discount.

The cost of pomalidomide is £8884 per 21-tablet pack (excluding tax). The average cost of a course of treatment is £44,420 (excluding tax).

The discount Celgene will provide to the NHS is confidential.

NICE’s final appraisal determination on pomalidomide is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

The final guidance is expected in January 2017. Once NICE issues a final guidance on pomalidomide, the NHS must make the drug available within 3 months.

Pomalidomide (Pomalyst)

Photo from Business Wire

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that pomalidomide be made available through the National Health Service (NHS).

NICE is recommending pomalidomide be available for use in combination with low-dose dexamethasone to treat adults with multiple myeloma who have received at least 3 previous treatments, including lenalidomide and bortezomib.

NICE previously evaluated pomalidomide in 2015 and said it could not recommend the drug, as analyses suggested pomalidomide doesn’t provide enough benefit to justify its high price.

Since that time, a committee advising NICE has reviewed additional data on pomalidomide.

And Celgene, the company that makes pomalidomide, has agreed to provide the NHS with a discount.

The cost of pomalidomide is £8884 per 21-tablet pack (excluding tax). The average cost of a course of treatment is £44,420 (excluding tax).

The discount Celgene will provide to the NHS is confidential.

NICE’s final appraisal determination on pomalidomide is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

The final guidance is expected in January 2017. Once NICE issues a final guidance on pomalidomide, the NHS must make the drug available within 3 months.

Pomalidomide (Pomalyst)

Photo from Business Wire

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that pomalidomide be made available through the National Health Service (NHS).

NICE is recommending pomalidomide be available for use in combination with low-dose dexamethasone to treat adults with multiple myeloma who have received at least 3 previous treatments, including lenalidomide and bortezomib.

NICE previously evaluated pomalidomide in 2015 and said it could not recommend the drug, as analyses suggested pomalidomide doesn’t provide enough benefit to justify its high price.

Since that time, a committee advising NICE has reviewed additional data on pomalidomide.

And Celgene, the company that makes pomalidomide, has agreed to provide the NHS with a discount.

The cost of pomalidomide is £8884 per 21-tablet pack (excluding tax). The average cost of a course of treatment is £44,420 (excluding tax).

The discount Celgene will provide to the NHS is confidential.

NICE’s final appraisal determination on pomalidomide is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

The final guidance is expected in January 2017. Once NICE issues a final guidance on pomalidomide, the NHS must make the drug available within 3 months.

Ibrutinib (Imbruvica)

Photo courtesy of Janssen

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that ibrutinib come off the Cancer Drugs Fund (CDF) and be made available through the National Health Service (NHS).

Ibrutinib is a Bruton’s tyrosine kinase inhibitor approved by the European Commission treat patients with chronic lymphocytic leukemia (CLL).

NICE is recommending that ibrutinib be made available through the NHS for previously treated CLL patients and untreated CLL patients who have 17p deletion or TP53 mutation.

This means patients will no longer have to apply to the CDF to obtain ibrutinib. The CDF is money the English government sets aside to pay for cancer drugs that haven’t been approved by NICE and aren’t available within the NHS.

Though certain NICE products and services are provided to Wales, Scotland, and Northern Ireland, the governments of these countries do not have a CDF

or similar program.

Following the decision to reform the CDF earlier this year, NICE began to reappraise all drugs in the CDF in April.

New recommendation

NICE previously said it could not recommend ibrutinib for routine NHS use. However, Janssen, the company that makes ibrutinib, agreed to reduce the

price of the drug for the NHS. Because of the discount, an independent appraisal committee was able to deem ibrutinib cost-effective.

The list price for a single tablet of ibrutinib (140 mg) is £51.10 (excluding tax). The cost of a year’s course of ibrutinib treatment is £55,954.50 (excluding tax).

The discount the NHS will receive is confidential. The Department of Health said the cost of ibrutinib will not constitute an excessive administrative burden on the NHS.

NICE’s final appraisal determination on ibrutinib is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

Once NICE issues a final guidance on ibrutinib, the NHS must make the drug available within 3 months.

Ibrutinib (Imbruvica)

Photo courtesy of Janssen

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that ibrutinib come off the Cancer Drugs Fund (CDF) and be made available through the National Health Service (NHS).

Ibrutinib is a Bruton’s tyrosine kinase inhibitor approved by the European Commission treat patients with chronic lymphocytic leukemia (CLL).

NICE is recommending that ibrutinib be made available through the NHS for previously treated CLL patients and untreated CLL patients who have 17p deletion or TP53 mutation.

This means patients will no longer have to apply to the CDF to obtain ibrutinib. The CDF is money the English government sets aside to pay for cancer drugs that haven’t been approved by NICE and aren’t available within the NHS.

Though certain NICE products and services are provided to Wales, Scotland, and Northern Ireland, the governments of these countries do not have a CDF

or similar program.

Following the decision to reform the CDF earlier this year, NICE began to reappraise all drugs in the CDF in April.

New recommendation

NICE previously said it could not recommend ibrutinib for routine NHS use. However, Janssen, the company that makes ibrutinib, agreed to reduce the

price of the drug for the NHS. Because of the discount, an independent appraisal committee was able to deem ibrutinib cost-effective.

The list price for a single tablet of ibrutinib (140 mg) is £51.10 (excluding tax). The cost of a year’s course of ibrutinib treatment is £55,954.50 (excluding tax).

The discount the NHS will receive is confidential. The Department of Health said the cost of ibrutinib will not constitute an excessive administrative burden on the NHS.

NICE’s final appraisal determination on ibrutinib is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

Once NICE issues a final guidance on ibrutinib, the NHS must make the drug available within 3 months.

Ibrutinib (Imbruvica)

Photo courtesy of Janssen

The National Institute for Health and Care Excellence (NICE) has issued a final appraisal determination recommending that ibrutinib come off the Cancer Drugs Fund (CDF) and be made available through the National Health Service (NHS).

Ibrutinib is a Bruton’s tyrosine kinase inhibitor approved by the European Commission treat patients with chronic lymphocytic leukemia (CLL).

NICE is recommending that ibrutinib be made available through the NHS for previously treated CLL patients and untreated CLL patients who have 17p deletion or TP53 mutation.

This means patients will no longer have to apply to the CDF to obtain ibrutinib. The CDF is money the English government sets aside to pay for cancer drugs that haven’t been approved by NICE and aren’t available within the NHS.

Though certain NICE products and services are provided to Wales, Scotland, and Northern Ireland, the governments of these countries do not have a CDF

or similar program.

Following the decision to reform the CDF earlier this year, NICE began to reappraise all drugs in the CDF in April.

New recommendation

NICE previously said it could not recommend ibrutinib for routine NHS use. However, Janssen, the company that makes ibrutinib, agreed to reduce the

price of the drug for the NHS. Because of the discount, an independent appraisal committee was able to deem ibrutinib cost-effective.

The list price for a single tablet of ibrutinib (140 mg) is £51.10 (excluding tax). The cost of a year’s course of ibrutinib treatment is £55,954.50 (excluding tax).

The discount the NHS will receive is confidential. The Department of Health said the cost of ibrutinib will not constitute an excessive administrative burden on the NHS.

NICE’s final appraisal determination on ibrutinib is now with consultees who have the opportunity to appeal against it. If there is no appeal, or an appeal is not upheld, the final appraisal determination is issued by NICE as a guidance.

Once NICE issues a final guidance on ibrutinib, the NHS must make the drug available within 3 months.