This is the current policy.
Policy Updates include:
Removed Qualchoice line of business (refer to CP.PCH.56)
Clarified age restriction to age to 4 years and < 6 years.
CP.PCH.56
Delandistrogene moxeparvovec-rokl (Elevidys)
7/15/2025
Policy Created.
Initial criteria
Elevidys is considered experimental and investigational and not medically necessary for the treatment of Duchenne muscular dystrophy patients at least 4 years of age who are ambulatory or non-ambulatory for the following reasons:
Elevidys does not have proven efficacy in the treatment of Duchenne muscular dystrophy for ambulatory patients.
The phase III EMBARK [NCT05096221] confirmatory trial, which evaluated patients aged 4 to 7 years, failed to meet its statistical primary endpoint of improvement versus placebo in the North Star Ambulatory Assessment (NSAA) total score.
Study 102 Part 1 [NCT03769116], which evaluated patients aged 4 years to 7 years, failed to demonstrate a statistically significant change in NSAA from baseline to week 48 after treatment. Data showed no clear association between expression of Elevidys micro-dystrophin and change in NSAA total score.
Elevidys does not have proven efficacy in the treatment of Duchenne muscular dystrophy for non-ambulatory patients.
The phase I ENDEAVOR - Study 103 [NCT04626674] was the only study that contained data on non-ambulatory patients with Duchenne muscular dystrophy. Study 103 was not designed to demonstrate clinical efficacy and there was no data to support effectiveness for non-ambulatory patients with Duchenne muscular dystrophy.
For questions about QualChoice Medical Coverage Policies, please contact your Provider Relations Representative at 800.235.7111 or 501.228.7111, Monday through Friday, 8:00 a.m. to 5:00 p.m.
