Sarepta continued to progress toward potential traditional approval for VYONDYS and AMONDYS, completing its confirmatory study while demonstrating a significant, albeit non-statistically significant, treatment benefit in slowing disease progression in Duchenne muscular dystrophy patients.

• The confirmatory study completed for VYONDYS and AMONDYS showed a meaningful treatment benefit, with a 30% slowing of disease progression when excluding COVID-19 affected participants.
• Despite missing statistical significance, subgroup analyses indicated strong benefits for treatment among likely progressors.
• Continued high patient compliance rates above 90%, despite the demanding infusion protocol, underscored the perceived value of the therapies.
• Sarepta plans to engage with the FDA to discuss transitioning from accelerated to traditional approval based on the totality of available evidence.