Sarepta Confident Of Full US Approval For Duchenne Gene Therapy By Mid-2024

Elevidys has enjoyed a strong launch, despite being restricted to a small number of Duchenne patients. Sarepta is hoping the US regulator will overlook trial problems and allow its expanded use.

Sarepta building

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...

Scrip Podcast

Keep up with the top pharma business news of the week. On the go.

Scrip brings you a weekly bulletin of top picks.

More from Business

More from Scrip