Exclusive: Sarepta's Elevidys has 'arduous' path back to market, FDA official says

The remarks suggest the license for the Duchenne muscular dystrophy gene therapy could be revoked over safety concerns.

Log In    Subscribe Exclusive   Sarepta Therapeutics' Duchenne therapy faces 'arduous' path back to market, senior FDA official says By Adam Feuerstein and Jason Mast Andrew Harnik/AP Sarepta Therapeutics, the maker of a gene therapy for Duchenne muscular dystrophy that is being temporarily shelved because of safety concerns, faces an "arduous and treacherous path" to try to get it back onto the market, a senior FDA official told STAT, suggesting the treatment's license could be revoked.    The official, who spoke to STAT on condition of anonymity, did not rule out the possibility that the therapy, Elevidys, could eventually be brought back on the market. Informed of the comments, a Sarepta spokesperson told STAT the company had not been given the same message by its agency contacts. READ MORE Sign up for Biotech Scorecard Unfiltered, uncompromising analysis on the biotech world. Learn more. In Case You Missed It 1   In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy By Adam Feuerstein and Jason Mast 2   The crisis over Sarepta's Duchenne therapy is a mess. But the lesson is clear By Matthew Herper 3   Replimune skin cancer therapy rejected by FDA, a sign of regulators' hardened stance By Adam Feuerstein 4   Drugmakers are racing to help patients stay awake — and could also make billions By Elaine Chen Don't miss out on another STAT investigation Unlock the world of health and medicine with a subscription to STAT+ Learn more STAT, 1 Exchange Place, Boston, MA ©2025, All Rights Reserved. I no longer wish to receive STAT emails Update Newsletter Preferences • Contact Us • Advertise with Us