Overview

• A senior FDA official described the path to market for Elevidys as “arduous and treacherous” and warned its license could be revoked.
• Officials say Sarepta would struggle to prove that enhanced liver-monitoring protocols can fully mitigate the acute liver-injury risks linked to two patient fatalities.
• Roche has voluntarily paused new Elevidys orders in countries that depend on FDA decisions but continues limited supply to ambulatory Duchenne patients in Brazil and Japan.
• Elevidys received accelerated approval in June 2024 despite failing to meet primary efficacy endpoints and treated over 900 patients before U.S. and other FDA-linked shipments were halted.
• Debate over the therapy’s benefit-risk balance has intensified as families and advocacy groups press for access while regulators demand robust safety evidence.