Sarepta has reiterated its belief that its gene therapy Elevidys can gain a full US approval across all patients with Duchenne muscular dystrophy in 2024, despite falling short in a pivotal study last year.
Elevidys (delandistrogene moxeparvovec-rokl) won an accelerated US Food and Drug Administration approval in June to treat ambulatory patients ages four to five years old with a confirmed Duchenne gene mutation. This was based on Phase II studies, but its Phase III EMBARK confirmatory trial missed its primary endpoint in late October
Key Takeaways
-
Sarepta’s gene therapy racked up $200m in sales in six months, despite being restricted to a small group of Duchenne patients
-
Analysts had feared that the Phase III trial miss would mean the FDA would not grant full approval in a broader group of patients
Read the full article – start your free trial today!
Join thousands of industry professionals who rely on Scrip for daily insights
- Start your 7-day free trial
- Explore trusted news, analysis, and insights
- Access comprehensive global coverage
- Enjoy instant access – no credit card required
Already a subscriber?
