Sarepta Therapeutics Surges After FDA Expands Approval for DMD Drug ELEVIDYS

Sarepta Therapeutics (NASDAQ), a leader in precision genetic medicine for rare diseases, saw a significant rise in after-hours trading on Thursday following the extended approval of its drug, ELEVIDYS, by the US Food and Drug Administration (FDA).

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Sarepta Therapeutics

ELEVIDYS is now approved for use in patients aged 4 and above with Duchenne muscular dystrophy (DMD) who have a specific gene mutation. The FDA has granted full approval for its use in ambulatory DMD patients and conditional approval for non-ambulatory patients, pending further research to confirm the drug’s benefits. ELEVIDYS is not approved for patients with certain gene deletions.

Sarepta’s CEO, Doug Ingram, and Dr. Jerry Mendell, the co-inventor of ELEVIDYS, expressed their enthusiasm about the expanded approval and its implications for the DMD community.

Ingram stated, “The expansion of the ELEVIDYS label to treat Duchenne patients is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science.”

As part of its agreement with the FDA, Sarepta will conduct a detailed study, named ENVISION, to further validate the benefits of ELEVIDYS for non-ambulatory DMD patients. This study is already in progress.

Additionally, Sarepta is collaborating with Roche to make ELEVIDYS accessible to patients worldwide. Sarepta will oversee the drug’s approval and sales in the U.S., while Roche will manage its approval and distribution in other countries.