Drugs

FDA Defers Action on Amicus BLA

FDA has deferred action on an Amicus Therapeutics BLA for cipaglucosidase alfa, the biological component of AT-GAA in late-onset Pompe disease, due to the agency’s inability to conduct a required manufacturing site inspection in China before the PDUFA action date. A company announcement says the inability to conduct the inspection was the only reason cited by FDA for the deferred action. No anticipated new action date was given, Amicus says.

AT-GAA is described as an investigational two-component therapy consisting of cipaglucosidase alfa, a unique recombinant human acid alpha-glucosidase enzyme with optimized carbohydrate structures, administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa. FDA granted it breakthrough therapy designation to treat late-onset Pompe disease based on clinical efficacy results from a Phase 1/2 clinical study.

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