Eteplirsen (Exondys 51)

Exondys 51 (eteplirsen) is limited to Duchenne muscular dystrophy patients with a confirmed DMD gene mutation amenable to exon 51 skipping and is not indicated for other DMD mutations; because clinical benefit has not been established, approval is not recommended. Requests must include genetic test confirmation of an exon 51–amenable mutation, documentation of a DMD diagnosis, and evidence the patient meets the policy’s coverage and safety criteria (continued approval contingent on confirmatory trial verification).

"Exondys 51 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 51 skipping."

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