FDA Expands Approval of Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy to Include Older Boys and Non-Ambulatory Patients

1. Expanded Approval: The FDA has broadened the approval of Sarepta's gene therapy, Elevidys, to treat Duchenne muscular dystrophy (DMD) in patients aged 4 and above, regardless of ambulatory status.
2. Traditional Approval: Elevidys received traditional approval for ambulatory patients aged 4 and older with a confirmed mutation in the DMD gene.
3. Accelerated Approval: The FDA granted accelerated approval for non-ambulatory patients aged 4 and older with a confirmed mutation in the DMD gene, contingent upon verification of clinical benefit in a confirmatory trial.
4. Disease Impact: DMD is a rare and serious genetic condition that worsens over time, leading to muscle weakness and wasting, with symptoms typically beginning in childhood.
5. Market Impact: Sarepta's stock surged by 34% after the FDA's decision, signaling strong market potential for the gene therapy.
6. Competitive Landscape: The DMD field is highly competitive, with companies like Regenxbio and Solid Biosciences advancing their gene therapy candidates.
7. Future Studies: Sarepta is conducting the ENVISION study to fulfill regulatory requirements for Elevidys globally and to verify clinical benefit in non-ambulatory patients.