Biologics

FDA Wins as Sarepta Folds and Stops Shipping Elevidys

FDA has won its regulatory battle (see earlier story) against gene therapy maker Sarepta Therapeutics. Late last evening (7/21) the company announced that it will agree to FDA’s request to pause all shipments of Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy 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,” it says.

On 7/18, FDA asked Sarepta to voluntarily halt Elevidys shipments in response to two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male Duchenne patients (see earlier story). Added pressure had mounted after Sarepta reported a third gene therapy patient death, which it says was related to a different therapy called SRP-9004, a clinical-stage therapy that is intended to treat a different disease (Limb-girdle muscular dystrophy Type 2D).

In response, Sarepta defiantly said it would initially continue shipping the gene therapy for exclusive use in ambulatory patients. This has now changed and the company has agreed to the agency’s request to cease shipping the product. In announcing its reversal, Sarepta said: “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process.”

Meanwhile, FDA has placed Sarepta’s investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold, and it revoked its platform technology designation. “The three deaths appear to have been a result of acute liver failure in individuals treated with Elevidys or investigational gene therapy using the same AAVrh74 serotype that is used in Elevidys,” an FDA notice says. “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits.”

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