First gene therapy for certain DMD patients gains FDA nod

The US Food and Drug Administration (FDA) yesterday approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients aged four through five years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.

The FDA granted accelerated approval of Elevidys (previously known as SRP-9001) to Sarepta Therapeutics (Nasdaq: SRPT) and may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Elevidy is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

Sarepta’s shares closed down 2.3% on Thursday and fell a further 3.9% to $120.00 pre-market today, on investor concerns about the more limited patient population that can be prescribed the drug than had been hoped.