Drugs

Stealth Wins Approval for Barth Syndrome Drug

FDA has granted accelerated approval to a Stealth Biotherapeutics NDA for Forzinity (elamipretide) injection for treating Barth syndrome, a rare and often fatal mitochondrial disease that primarily affects boys. The disease typically begins with severe heart failure in infancy, significantly reducing life expectancy and impairing quality of life, an agency release says. Patients who reach adolescence or adulthood typically face chronic fatigue, low stamina, and exercise intolerance.

FDA says Forzinity is designed to improve mitochondrial structure and function by binding to the inner mitochondrial membrane. Its approval was based on data showing that the drug improved the strength of the quadriceps muscle, which the agency deemed reasonably likely to predict meaningful patient benefits, such as improved ability to stand or walk.

Under FDA’s accelerated pathway, Stealth Biotherapeutics must conduct a confirmatory trial to verify that muscle strength gains lead to functional improvements in patients, FDA notes.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome