SRPT - FDA Approves Expanded Use For Sarepta's Rare Muscular Dystrophy Gene Therapy | Benzinga
Thursday, the FDA approved labeled indication for Sarepta Therapeutics Inc’s (NASDAQ:SRPT) Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.
Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients (patients who can walk) and accelerated approval for non-ambulatory patients (patients who cannot walk).
Related: Sarepta Downgraded As FDA Decision Date Approaches, Shares Up 50% Over Last Six Months.
Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD ...