Sarepta’s Gene Therapy Benefits Are Unclear, FDA Staff Says

Sarepta Therapeutics Inc.’s experimental gene therapy for a debilitating muscle disease that mainly affects boys doesn’t clearly show benefit and raises safety concerns, Food and Drug Administration staff said in documents posted ahead of a review by advisers from outside the agency.

Studies have failed to show unambiguous evidence that Sarepta’s candidate treatment for Duchenne muscular dystrophy will likely benefit patients, FDA staff said in a document published Wednesday ahead of the FDA advisory panel meeting scheduled for Friday. The FDA reviewers also voiced concern about potential safety issues with giving people a gene therapy if it turns out to be ineffective. If approved, Sarepta’s SRP-9001 would become the first gene therapy to treat muscular dystrophy.