Investing.com – Sarepta Therapeutics (NASDAQ:), a pioneer in genetic precision medicine for rare diseases, saw a surge in deals Thursday after receiving expanded approval from the U.S. Food and Drug Administration (FDA) for its drug ELEVIDYS.
The drug can now be used for patients aged 4 years and older who suffer from Duchenne muscular dystrophy (DMD), a specific gene mutation.
The FDA has given full approval to ELEVIDIS for patients with DMD who can walk and conditional approval for patients who cannot walk. Continued approval for patients unable to walk will depend on further studies confirming the drug’s benefits. ELEVIDIS should not be used in patients with deletions of certain genes.
Sarepta CEO Doug Ingram and Dr. Jerry Mendell, co-creator of ELEVIDYS, expressed their excitement and hope for what this expanded approval means for the DMD community.
Ingram said, “Expanding the ELEVIDYS brand to treat Duchenne patients is a defining moment for the Duchenne community. Today is also a watershed moment for the promise of gene therapy and the triumph of science.”
As part of the agreement with the FDA, Sarepta will conduct a detailed study to further confirm the benefits of ELEVIDYS for patients with DMD who are unable to walk. A study called ENVISION is already underway.
Sarepta is also working with fellow company Roche to bring ELEVIDYS to patients around the world. Sarepta will handle approval and sales of the drug in the United States, while Roche will handle approval and distribution in other countries.