Golodirsen (Vyondys 53®)

Golodirsen (Vyondys 53) is FDA‑approved under accelerated approval for DMD patients with a confirmed DMD gene mutation amenable to exon 53 skipping, but clinical benefit has not been established and this policy does not recommend approval due to lack of clinical efficacy data. Coverage requires documentation of the confirmed exon‑53‑amenable DMD mutation, a diagnosis of DMD, adherence to specified coverage and safety criteria, and acknowledgement that continued approval depends on confirmatory trials.

"Vyondys 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping."

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