FDA approves Sarepta Therapeutics’ gene therapy
Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced US Food and Drug Administration (FDA) accelerated approval of Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
This indication is approved under accelerated approval based on expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.
Elevidys is the first FDA approved gene therapy to treat Duchenne muscular dystrophy.
