Viltepso® (viltolarsen)

Viltepso (viltolarsen) is FDA‑approved for Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 53 skipping, but this policy does not recommend approval because clinical benefit has not been established and efficacy data are lacking. Coverage would require documentation of the confirmed exon‑53‑amenable mutation and that the request meets the FDA‑approved indication or compendial use and applicable (unspecified) coverage/safety criteria, noting continued FDA accelerated approval is contingent on confirmatory trials.

"Viltepso 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."

Sign up to see full coverage criteria, indications, and limitations.