A senior FDA official warns that Sarepta Therapeutics, maker of a gene therapy for Duchenne muscular dystrophy, faces a challenging path to get its treatment back on the market due to safety concerns. The official suggests that the company’s license could be revoked if it fails to prove new safety protocols can eliminate the risk of liver injuries linked to patient deaths.
The official notes that proving safety is difficult when an issue has already been proven not safe, raising questions about how Sarepta can demonstrate its treatment’s safety. This hurdle makes it a daunting task for the company to overcome and bring Elevidys back onto the market without risking further regulatory action.
Source: https://www.statnews.com/2025/07/22/fda-sarepta-elevidys-muscular-dystrophy-drug