FDA Approves Expanded Use For Sarepta's Rare Muscular Dystrophy Gene Therapy

Loading... 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). 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 gene. Consistent with the accelerated approval pathway, Sarepta will ...