London: The US Food and Drug Administration Extended use Of Sarepta Gene Therapy of Therapeutics Patients with Duchenne muscular dystrophy More than 100 children aged four and above died on Thursday, sending the company’s shares soaring 36 per cent in extended trading.
The agency granted traditional approval to patients four years of age and older who can walk, as well as Quick Approval For those who can’t.
The FDA granted accelerated approval to the therapy in June last year — the first of its kind for DMD in patients four to five years old who can walk. Accelerated approval is a process that allows the agency to give the green light to a treatment even before there is confirmatory data showing it is effective.
Continued approval for the use of this therapy in Duchenne patients who cannot walk may depend on verification of clinical benefit in a confirmatory trial.
However, the company said the therapy, called Alevidis, should not be used in patients with certain mutations in the DMD gene.
(Reporting by Sriparna Roy and Leroy Leo in Bengaluru; Editing by Shailesh Kuber, Alan Barona and Pooja Desai)
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