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First Brain-Injected Gene Therapy Approved by FDA
The US Food and Drug Administration (FDA) has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, marketed under the brand name Kebilidi, is the first in the United States to be injected directly into the brain. It is approved for children with fully developed skulls and for adults.
AADC is an enzyme that helps the body make dopamine. AADC deficiency affects patients’ physical, mental, and behavioral health from infancy, leading to severe disabilities and shorter lifespan. Children with AADC may also experience painful seizure-like episodes called oculogyric crises.
Kebilidi (generic name: eladocagene exuparvovec) is injected into a specific area of the brain where it boosts AADC, restoring dopamine production and gradually improving movement-related symptoms. This surgery is to be performed only by brain surgeons in specialized centers.
The FDA approval was based on the therapy’s safety and effectiveness as shown in an ongoing clinical trial involving 13 children diagnosed with AADC deficiency. According to PTC Therapeutics, the maker of Kebilidi, long-term follow-up studies of the participants are still needed, and additional proof of the therapy’s benefits are required for full FDA approval.
Common side effects of Kebilidi therapy may include involuntary movements (dyskinesia), anemia, fever, low blood pressure, excessive salivation, problems sleeping, low blood levels of certain minerals, and complications after the injection, including breathing or heart problems. The surgical procedure for injecting Kebilidi also carries certain risks, such as cerebrospinal fluid leaks, bleeding in the brain, inflammation, strokes, and infections.
A version of this article appeared on WebMD.com.
The US Food and Drug Administration (FDA) has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, marketed under the brand name Kebilidi, is the first in the United States to be injected directly into the brain. It is approved for children with fully developed skulls and for adults.
AADC is an enzyme that helps the body make dopamine. AADC deficiency affects patients’ physical, mental, and behavioral health from infancy, leading to severe disabilities and shorter lifespan. Children with AADC may also experience painful seizure-like episodes called oculogyric crises.
Kebilidi (generic name: eladocagene exuparvovec) is injected into a specific area of the brain where it boosts AADC, restoring dopamine production and gradually improving movement-related symptoms. This surgery is to be performed only by brain surgeons in specialized centers.
The FDA approval was based on the therapy’s safety and effectiveness as shown in an ongoing clinical trial involving 13 children diagnosed with AADC deficiency. According to PTC Therapeutics, the maker of Kebilidi, long-term follow-up studies of the participants are still needed, and additional proof of the therapy’s benefits are required for full FDA approval.
Common side effects of Kebilidi therapy may include involuntary movements (dyskinesia), anemia, fever, low blood pressure, excessive salivation, problems sleeping, low blood levels of certain minerals, and complications after the injection, including breathing or heart problems. The surgical procedure for injecting Kebilidi also carries certain risks, such as cerebrospinal fluid leaks, bleeding in the brain, inflammation, strokes, and infections.
A version of this article appeared on WebMD.com.
The US Food and Drug Administration (FDA) has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, marketed under the brand name Kebilidi, is the first in the United States to be injected directly into the brain. It is approved for children with fully developed skulls and for adults.
AADC is an enzyme that helps the body make dopamine. AADC deficiency affects patients’ physical, mental, and behavioral health from infancy, leading to severe disabilities and shorter lifespan. Children with AADC may also experience painful seizure-like episodes called oculogyric crises.
Kebilidi (generic name: eladocagene exuparvovec) is injected into a specific area of the brain where it boosts AADC, restoring dopamine production and gradually improving movement-related symptoms. This surgery is to be performed only by brain surgeons in specialized centers.
The FDA approval was based on the therapy’s safety and effectiveness as shown in an ongoing clinical trial involving 13 children diagnosed with AADC deficiency. According to PTC Therapeutics, the maker of Kebilidi, long-term follow-up studies of the participants are still needed, and additional proof of the therapy’s benefits are required for full FDA approval.
Common side effects of Kebilidi therapy may include involuntary movements (dyskinesia), anemia, fever, low blood pressure, excessive salivation, problems sleeping, low blood levels of certain minerals, and complications after the injection, including breathing or heart problems. The surgical procedure for injecting Kebilidi also carries certain risks, such as cerebrospinal fluid leaks, bleeding in the brain, inflammation, strokes, and infections.
A version of this article appeared on WebMD.com.
FDA OKs New Drug for Urinary Tract Infections
The Food and Drug Administration (FDA) has approved Orlynvah, a new oral treatment for uncomplicated urinary tract infections (UTIs) in women who have limited options for effective antibiotic therapy.
Uncomplicated UTIs are bladder infections that typically affect women who don’t have other issues like kidney disease or urinary tract abnormalities. These infections are common, affecting around half of all women at least once in their lives.
Treating UTIs can be difficult when standard antibiotics don’t work well, often because of antibiotic resistance or certain health conditions. Orlynvah offers a promising new option by combining two drugs, sulopenem etzadroxil and probenecid, in one oral tablet. This combination helps keep the antibiotic in the body longer, making it work better, especially against bacteria that resist traditional treatments. Orlynvah is specifically approved to target infections from bacteria like Escherichia coli, Klebsiella pneumoniae, and Proteus mirabilis, which can be harder to treat.
Marjorie Golden, MD, an infectious disease specialist at Yale New Haven Hospital in Connecticut, described Orlynvah as a much-needed alternative for women struggling with difficult-to-treat UTIs.
“Orlynvah has the potential to be an important treatment option for those who need it,” she said in a news release from Iterum Therapeutics, the drug’s maker.
The FDA approved Orlynvah based on two large clinical trials involving over 3,800 women. In these studies, Orlynvah worked as well as or better than antibiotics like ciprofloxacin and Augmentin. It was generally well-tolerated, though common side effects included diarrhea, nausea, yeast infections, and headaches.
The FDA advises people to discuss their medical history with their doctor before taking Orlynvah, especially if they have conditions like gout, kidney stones, or allergies to other antibiotics.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration (FDA) has approved Orlynvah, a new oral treatment for uncomplicated urinary tract infections (UTIs) in women who have limited options for effective antibiotic therapy.
Uncomplicated UTIs are bladder infections that typically affect women who don’t have other issues like kidney disease or urinary tract abnormalities. These infections are common, affecting around half of all women at least once in their lives.
Treating UTIs can be difficult when standard antibiotics don’t work well, often because of antibiotic resistance or certain health conditions. Orlynvah offers a promising new option by combining two drugs, sulopenem etzadroxil and probenecid, in one oral tablet. This combination helps keep the antibiotic in the body longer, making it work better, especially against bacteria that resist traditional treatments. Orlynvah is specifically approved to target infections from bacteria like Escherichia coli, Klebsiella pneumoniae, and Proteus mirabilis, which can be harder to treat.
Marjorie Golden, MD, an infectious disease specialist at Yale New Haven Hospital in Connecticut, described Orlynvah as a much-needed alternative for women struggling with difficult-to-treat UTIs.
“Orlynvah has the potential to be an important treatment option for those who need it,” she said in a news release from Iterum Therapeutics, the drug’s maker.
The FDA approved Orlynvah based on two large clinical trials involving over 3,800 women. In these studies, Orlynvah worked as well as or better than antibiotics like ciprofloxacin and Augmentin. It was generally well-tolerated, though common side effects included diarrhea, nausea, yeast infections, and headaches.
The FDA advises people to discuss their medical history with their doctor before taking Orlynvah, especially if they have conditions like gout, kidney stones, or allergies to other antibiotics.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration (FDA) has approved Orlynvah, a new oral treatment for uncomplicated urinary tract infections (UTIs) in women who have limited options for effective antibiotic therapy.
Uncomplicated UTIs are bladder infections that typically affect women who don’t have other issues like kidney disease or urinary tract abnormalities. These infections are common, affecting around half of all women at least once in their lives.
Treating UTIs can be difficult when standard antibiotics don’t work well, often because of antibiotic resistance or certain health conditions. Orlynvah offers a promising new option by combining two drugs, sulopenem etzadroxil and probenecid, in one oral tablet. This combination helps keep the antibiotic in the body longer, making it work better, especially against bacteria that resist traditional treatments. Orlynvah is specifically approved to target infections from bacteria like Escherichia coli, Klebsiella pneumoniae, and Proteus mirabilis, which can be harder to treat.
Marjorie Golden, MD, an infectious disease specialist at Yale New Haven Hospital in Connecticut, described Orlynvah as a much-needed alternative for women struggling with difficult-to-treat UTIs.
“Orlynvah has the potential to be an important treatment option for those who need it,” she said in a news release from Iterum Therapeutics, the drug’s maker.
The FDA approved Orlynvah based on two large clinical trials involving over 3,800 women. In these studies, Orlynvah worked as well as or better than antibiotics like ciprofloxacin and Augmentin. It was generally well-tolerated, though common side effects included diarrhea, nausea, yeast infections, and headaches.
The FDA advises people to discuss their medical history with their doctor before taking Orlynvah, especially if they have conditions like gout, kidney stones, or allergies to other antibiotics.
A version of this article first appeared on Medscape.com.