Investing.com - Sarepta Therapeutics (NASDAQ:SRPT), a pioneer in precision genetic medicine for rare diseases, surged sharply following the market open on Friday after receiving extended approval from the US Food and Drug Administration (FDA) for its drug, ELEVIDYS.
The drug can now be used for patients aged 4 and above who have Duchenne muscular dystrophy (DMD), a specific gene mutation.
The FDA has given full approval for the use of ELEVIDYS for DMD patients who can walk, and conditional approval for patients who cannot walk. The continuation of the approval for non-walking patients will depend on further research confirming the drug's benefits. ELEVIDYS cannot be used in patients with particular gene deletions.
"Net-net, this was the home-run scenario that was largely non-consensus given most had expected full approval in ambulatory patients," Piper Sandler analysts said in their comments on the news, reiterating their Overweight rating on the stock.
Sarepta shares soared 32% at the market open.
Sarepta's CEO, Doug Ingram, and Dr. Jerry Mendell, the co-inventor of ELEVIDYS, both expressed their excitement and hope for what this expanded approval means for the DMD community.
Ingram said, "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 carry out a detailed study to further confirm the benefits of ELEVIDYS for non-walking DMD patients. The study, called ENVISION, is already underway.
Sarepta is also working with another company, Roche, to bring ELEVIDYS to patients worldwide. Sarepta will handle the drug's approval and sale in the U.S., while Roche will take care of its approval and distribution in other countries.