Elevidys DMD gene therapy now FDA-approved for ages 4 and older

Label expansion will make one-time treatment available to more patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), authorizing the one-time gene therapy for individuals with Duchenne muscular dystrophy (DMD) ages 4 and older regardless of their ability to walk. Previously, the treatment had been approved for DMD children ages 4 to 5 who could walk.

Doug Ingram, president and CEO of Elevidys’ developer Sarepta Therapeutics, said the expanded approval “is a defining moment for the Duchenne community” as well as “a watershed occasion for the promise of gene therapy and a win for science,” in a company press release.

The move was welcomed with excitement by the muscular dystrophy community.

“We are delighted that the FDA has expanded the label for Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy to include ambulatory and non-ambulatory Duchenne patients who are older than 4 years of age,” Debra Miller, founder and CEO of CureDuchenne, said in a statement to Muscular Dystrophy News.

Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD), said the FDA’s decision “represents a monumental leap forward in our collective efforts to end Duchenne muscular dystrophy.”

“By expanding the label’s age inclusion and removing ambulation restrictions, Elevidys can now reach and benefit a more diverse population of Duchenne patients,” Furlong said in a PPMD press release, adding “patients and families need treatment options, and we applaud the expansion of these options.”

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Duchenne is caused by mutations in the gene DMD, which result in virtually no production of dystrophin, a protein that normally helps to prevent damage in muscle cells. Lacking functional dystrophin, muscle cells of people with Duchenne accumulate atypical damage over time, which ultimately drives symptoms like muscle weakness and wasting.

Elevidys is designed to deliver a gene encoding micro-dystrophin, a shortened but functional version of the dystrophin protein, to muscle cells.

While more patients now are eligible for the therapy, it remains contraindicated, or not recommended for use, among individuals who have any deletion in exon 8 and/or exon 9 in the DMD gene. It also is not recommended for patients with high levels against AAVrh74, the viral vector that the therapy uses to deliver its genetic cargo to cells. Exons are the coding sections that have the information to produce proteins.

Almost exactly one year ago, the FDA granted Elevidys accelerated approval to treat children with DMD ages 4 to 5 who are able to walk. Accelerated approval is a mechanism that allows the FDA to authorize a therapy based on early clinical data suggesting that the treatment will likely benefit patients. As a condition of accelerated approval, drug developers need to conduct additional clinical testing to prove the treatment’s benefit.

With the new expansion, Elevidys now is indicated to treat patients ages 4 and older, irrespective of whether or not they are able to walk.

“The initial approval of Elevidys was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne,” said Jerry Mendell, MD, senior advisor of medical affairs at Sarepta and co-inventor of Elevidys. (DMD primarily affects boys and men, though Elevidys is approved to treat the condition irrespective of gender or biological sex.)

Sarepta offers a program called SareptAssist, which includes resources for financial assistance and preparing for treatments. The program can be contacted by phone at 1-888-727-3782.

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Treatment now to be available to DMD patients regardless of walking ability

The FDA’s expansion was supported largely by data from the Phase 3 EMBARK trial (NCT05096221), which tested the therapy against a placebo in more than 120 boys with DMD ages 4 to 7. Although the study missed its main goal, the results showed that Elevidys significantly outperformed the placebo in several key secondary measures of motor function — one being the time it took patients to walk 10 meters (about 33 feet) or rise from a lying position.

According to the FDA, these findings were “compelling and … indicate clinical benefit compared to [a] placebo,” leading the agency to grant full approval for Elevidys in DMD patients who can walk. The agency noted that common side effects of Elevidys include vomiting, nausea, acute liver injury, fever, and low platelet counts, called thrombocytopenia.

[This decision] marks a pivotal moment for the community, offering renewed hope and tangible progress in our fight against DMD.

Although Elevidys’ use in ambulatory (able-to-walk) patients is now traditionally approved, its use in nonambulatory patients is under accelerated approval pending additional data to confirm its benefit in this population. Sarepta is running a separate Phase 3 trial, dubbed ENVISION (NCT05881408), to test Elevidys in DMD patients who are older and/or nonambulatory.

Assuming that the results are positive, ENVISION is expected to serve as a confirmatory study to support full traditional FDA approval of Elevidys in DMD patients who cannot walk. ENVISION is still recruiting patients at sites in Belgium, Italy, Japan, Spain, and the U.K.

“The full approval for all ambulant patients and accelerated approval for all non-ambulant patients is not only a scientific accomplishment but a major source of hope for countless families affected by this relentless disease,” Barry Byrne, MD, PhD, chief medical advisor at the Muscular Dystrophy Association, said in a press release from the advocacy group.

Sharon Hesterlee, PhD, the association’s chief research officer, added that the approval “marks a pivotal moment for the community, offering renewed hope and tangible progress in our fight against DMD.”