FDA’s Peter Marks seems inclined to back Sarepta’s gene therapy

The Food and Drug Administration’s top regulator of gene therapies, Peter Marks, suggested granting some form of full approval to Elevidys, the Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy, based on his comments in a STAT event. He did not comment specifically on the ongoing FDA review of Elevidys. However, Marks described a hypothetical case that suggested the failure of a confirmatory study in October didn’t bother him much. He stated that the FDA has approved products based on a situation where the overall outcome didn’t come out perfectly. This implies that Elevidys may still receive some form of full approval despite the failure of the study.

Source link