Vyondys 53® (golodirsen) Injection

Vyondys 53 (golodirsen) is not recommended for approval/coverage due to lack of demonstrated clinical efficacy despite FDA accelerated approval for DMD patients with DMD gene mutations amenable to exon 53 skipping. If requested, coverage would require documentation of a confirmed DMD diagnosis, genetic test proof the mutation is amenable to exon 53 skipping, fulfillment of the policy’s specific coverage and safety criteria, and acknowledgment that 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."

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