Overview
- Topline Phase 3 ESSENCE results showed casimersen and golodirsen failed the primary goal, despite numerical trends favoring treatment.
- The nine-year study followed 225 boys aged 6 to 13 for 96 weeks using a four‑step climb measure that did not reach statistical significance.
- Sarepta said it will meet the FDA to discuss converting the drugs’ existing accelerated approvals to traditional approvals.
- The company cited pandemic disruptions to participation and data collection and highlighted post‑hoc analyses suggesting larger effects, which may draw skepticism.
- The setback intensifies scrutiny of Sarepta’s DMD portfolio after a July Elevidys market pause following three deaths, even as Q3 revenue of $399.4 million beat expectations.