Investing.com – Zarephath Therapeutics (NASDAQ:), a pioneer in precision genetic medicine for rare diseases, rose sharply after the market opened 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 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. Continued approval for non-running patients will depend on further research confirming the drug’s benefits. ELEVIDYS cannot be used in patients with certain gene deletions.
“Net-net, this was the at-home scenario that was largely non-consensus as most expected full approval in outpatients,” Piper Sandler analysts said in commenting on the news, reiterating their Overweight rating on the stock.
Shares of Sarepta rose 32% when the market was open.
Sarepta 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 marks a turning point for the promise of gene therapy and a victory for science.”
As part of the agreement with the FDA, Sarepta will conduct a detailed study to further confirm the benefits of ELEVIDYS for non-current DMD patients. The research, called ENVISION, is already underway.
Sarepta is also working with another company, Roche, to bring ELEVIDYS to patients worldwide. Sarepta will handle approval and sales of the drug in the US, while Roche will handle approval and distribution in other countries.
