Biologics

FDA to Demand New Elevidys Safety Data from Sarepta

FDA reportedly will require additional safety data from Sarepta Therapeutics before the agency considers supporting the continued use of the company’s Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec-rokl) following three recent patient deaths — two associated with Elevidys and the other associated with an experimental gene therapy for limb-girdle muscular dystrophy, which uses the same AAVrh74 viral vector as the DMD treatment. Its continued use in the U.S. market is contingent on data that will assure the agency that the therapy’s use will not result in more deaths, an unnamed FDA official told Bloomberg, adding that the company will have to produce a detailed plan as to how it can be safely reintroduced.

Earlier this week, Sarepta reluctantly agreed to an FDA request to pause all shipments of Elevidys in the U.S. “This proactive step will allow Sarepta the necessary time to respond to any requests for information and allow Sarepta and FDA to complete the Elevidys safety labeling supplement process,” the company said at the time.

The FDA official told Bloomberg that the agency has yet to see a convincing account as to why Elevidys was tied to the two liver failure fatal cases, adding that there may be no simple fix to the problem. There are no FDA employees who support Elevidys’s return to the market based on current data, Bloomberg reported.

Asked to confirm that FDA will require new clinical data before allowing Sarepta’s Elevidys back on the market, an HHS spokesperson said: “We are looking at all the tools we have.”

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

Read more

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