Golodirsen (Vyondys 53)

Golodirsen (Vyondys 53) is covered only for Duchenne muscular dystrophy patients with a confirmed DMD gene mutation amenable to exon 53 skipping; patients without an exon‑53‑amenable mutation are excluded. Initial approval requires a DMD diagnosis and genetic confirmation of an exon‑53‑amenable mutation, dosing is 30 mg/kg IV weekly, approval is for 12 months, and reauthorization requires documented clinical benefit (e.g., increased dystrophin or improved 6MWD) and meeting applicable safety criteria.

"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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