Golodirsen (Vyondys 53®)

Vyondys 53 (golodirsen) has FDA accelerated approval for treating Duchenne muscular dystrophy in patients with a DMD gene mutation amenable to exon 53 skipping, but Evicore does not recommend approval and the drug’s clinical benefit has not been established. Coverage requires a documented DMD diagnosis, genetic test confirming exon 53 amenability, adherence to applicable coverage and safety criteria, and continued approval may depend on confirmatory trial results.

"Due to lack of clinical efficacy data, approval is not recommended for Vyondys 53."

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