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Investing.com – Sarepta Shares of Therapeutics Inc. (NASDAQ:), a leader in precision genetic medicine for rare diseases, rose sharply after the market open on Friday after receiving expanded approval from the US Food and Drug Administration (FDA) for its drug ELEVIDYS.
The drug can now be used for patients aged 4 years and older with Duchenne muscular dystrophy (DMD), a specific genetic mutation.
The US Food and Drug Administration (FDA) has granted full approval for ELEVIDYS for use in DMD patients who can walk, and conditional approval for patients who cannot walk. Continued approval for patients who cannot walk will depend on more research confirming the drug’s benefits. ELEVIDYS cannot be used in patients with certain gene deletions.
“Net-net, this was a play-at-home scenario that was not very unanimous given that most expected full approval for ambulatory patients,” Piper Sandler analysts said in their comments on the news, reiterating their overweight rating on the stock.
Shares of Sarepta rose 32% at market open.
Sarepta CEO Doug Ingram and Dr. Jerry Mendel, co-inventor of ELEVIDYS, both expressed excitement and hope for what this expanded approval means for the DMD community.
“Expanding the ELEVIDYS label to treat Duchenne patients is a defining moment for the Duchenne community,” Ingram said. “Today also marks a watershed occasion for the promise of gene therapy and the triumph of science.”
As part of its agreement with the FDA, Sarepta will conduct a detailed study to further confirm the benefits of ELEVIDYS for DMD patients who are unable to walk. The study, called ENVISION, is already underway.
Sarepta is also working with another company, Roche, to bring ELEVIDYS to patients around the world. Sarepta will approve and sell the drug in the United States, while Roche will approve and distribute it in other countries.
