Exondys 51 (eteplirsen)

Section: Injections
Effective Date: December 01, 2019
Revised Date: November 26, 2019


Exondys 51 (eteplirsen) is an antisense oligonucleotide 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. The accelerated approval of Exondys 51 (eteplirsen) is based on a surrogate endpoint, but a clinical benefit, including improved motor function has not been established. Continued approval of this agent by the FDA is contingent upon demonstrating clinical benefit in an ongoing phase III study. 


Coverage is subject to the specific terms of the member's benefit plan. 

Federal Employee Program members (FEP) should check with their Retail Pharmacy Program to determine if prior approval is required by calling the Retail Pharmacy Program at 1-800-624-5060 (TTY: 1-800-624-5077). FEP members can also obtain the list through the www.fepblue.org website.

Exondys 51 (eteplirsen) is considered experimental/investigational and therefore non-covered as there is a lack of conclusive evidence confirming clinical efficacy.

Procedure Codes