Vyondys 53® (golodirsen)

Vyondys 53 (golodirsen) is FDA-approved under accelerated approval for treatment of DMD in patients with a confirmed DMD gene mutation amenable to exon 53 skipping, but EviCore does not recommend approval because clinical benefit has not been established. Key requirement: documentation of a confirmed mutation amenable to exon 53 skipping is required and continued approval is contingent on verification of clinical benefit in a confirmatory trial.

"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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