Golodirsen (Vyondys 53)

Evicore does not recommend approval/coverage of golodirsen (Vyondys 53) despite its FDA accelerated approval for DMD patients with DMD gene mutations amenable to exon 53 skipping, citing lack of established clinical benefit and insufficient efficacy data. If considered, use must be for the FDA‑approved indication with a confirmed exon 53–amenable DMD mutation and meet applicable coverage and safety criteria, and continued approval may depend on confirmatory trial results.

"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. This indication is app..."

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