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# Sarepta Seeking Gene Therapy Expanded Use
- URL: https://www.fdaweb.com/sarepta-seeking-gene-therapy-expanded-use/
- Published: 2023-10-30T12:00:00.000Z
- Updated: 2026-09-14T14:14:41.000Z
- Author: David McFarland
- Tags: FDA Policy/General, #legacy-id-D5155676

Sarepta Therapeutics says it will file a supplemental BLA for Elevidys (delandistrogene moxeparvovec-rokl) to expand the therapy’s use in all patients with Duchenne muscular dystrophy (DMD). In June, FDA granted the gene therapy accelerated approval to treat pediatric patients aged four and five years of age who have DMD with a confirmed mutation in the DMD gene and who do not have a pre-existing medical reason preventing treatment.

Sarepta says its decision to seek an expanded use is based on just-announced topline results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of Elevidys in patients with DMD between the ages of four through seven years. “The results of EMBARK, our double-blind, placebo-controlled trial, support the conclusion that Elevidys modifies the trajectory of Duchenne and benefits patients across age groups living with this ferociously degenerative disease,” the company says. “The results favored Elevidys across all endpoints in the study, including achieving statistical significance on all pre-specified key secondary endpoints and in each age subgroup of the key secondary endpoints. Indeed, passing five seconds on time to rise is the strongest predictor of early loss of ambulation and in EMBARK, Elevidys reduced those odds over 52 weeks by greater than 90%.”

Sarepta notes that in EMBARK participants treated with Elevidys showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, but the primary endpoint was not met. “In the study, Elevidys-treated patients improved 2.6 points on their North Star Ambulatory Assessment (NSAA) total score 52 weeks after treatment compared to 1.9 points in placebo-treated patients,” the company says. “The difference of 0.65 points between treated and placebo groups did not reach statistical significance (n=125; p=0.24).”

Elevidys’ original approval sparked controversy when CBER director **Peter Marks** overrode his Center’s review team ([see earlier story](https://fdaweb.com/login.php?sa=v&aid=D5154754&cate=&stid=%241%24i0%2F.Dt2.%24pXyTJ3MTgbA6dfJR7MlaS1&ref=fdaweb.com)), a move reminiscent of a review override ([see story](http://fdaweb.com/login.php?sa=v&aid=D5136852&searchWords=sarepta&cate=S&stid=%241%24nN1.kY4.%24eCp4eZGUWR02t7DKQHrRO.&ref=fdaweb.com)) almost seven years earlier by then-CDER director **Janet Woodcock** on a Sarepta NDA for DMD drug Exondys 51 (eteplirsen). Both cases add up to top-level FDA responsiveness to activists for a U.S. patient population estimated at about 870,000, mostly males under 25 who die of the disease by about that age.