This document addresses gene therapy for Duchenne muscular dystrophy (DMD), a rare and serious genetic disease affecting muscle strength and movement. Gene therapy is being proposed as a one-time treatment to significantly lessen the severity of DMD. At this time, one gene therapy has been approved by the Food and Drug Administration (FDA) to treat DMD: delandistrogene moxeparvovec-rokl (ELEVIDYS), an adeno-associated virus vector-based gene therapy.

Note: Please refer to the applicable clinical pharmacy criteria used by the Plan for information regarding disease-modifying treatments for DMD; for example: casimersen (Amondys 45), viltolarsen (Viltepso), and golodirsen (Vyondys 53).

Position Statement

Medically Necessary:

A one-time infusion of delandistrogene moxeparvovec-rokl (ELEVIDYS) is considered medically necessary in individuals who meet all of the following criteria:

1. Diagnosis of Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene; and
2. No deletion in exon 8 or exon 9 in DMD gene; and
3. Ambulatory; and
4. Age 4 through 5 years (at least 4 years 0 days and less than 6 years old); and
5. Anti-AAVrh74 total binding antibody titers less than 1:400; and
6. Absence of active infection; and
7. Absence of significant liver dysfunction or disease, defined as at least one of the following:
   1. Preexisting liver impairment; or
   2. Chronic hepatic condition; or
   3. Acute liver disease (e.g., acute hepatic viral infection).

Investigational and Not Medically Necessary:

Delandistrogene moxeparvovec-rokl is considered investigational and not medically necessary when the criteria above are not met.