FDA grants accelerated approval for Sarepta's ELEVIDYS gene therapy for Duchenne MD

Sarepta Therapeutics, Inc.

First FDA-approved gene therapy for Duchenne muscular dystrophy; for ambulatory patients aged 4-5.
Accelerated approval based on micro-dystrophin expression; confirmatory trial EMBARK reads out late 2023.
Contraindicated in patients with deletions in exon 8 and/or 9 of the DMD gene.
Common adverse reactions include vomiting, nausea, liver enzyme increases, pyrexia, thrombocytopenia.

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