RARE Daily

FDA Grants Sarepta Expanded Approval for DMD Gene Therapy

June 21, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Sarepta Therapeutics an expansion to the labeled indication for its Duchenne muscular dystrophy gene therapy Elevidys to include individuals with a confirmed mutation in the DMD gene who are at least 4 years of age.

The FDA granted traditional approval for ambulatory patients. The agency also granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. The expanded approval comes a year after the FDA first granted Elevidys accelerated approval for children with DMD ages 4 through 5.

Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

“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, co-inventor of Elevidys and senior advisor, Medical Affairs, Sarepta. “This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.”

DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms and other areas. Most patients require full-time use of a wheelchair in their early teens, and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.

Elevidys addresses the root genetic cause of Duchenne—mutations in the dystrophin gene that result in the lack of dystrophin protein—by delivering a gene that codes for a shortened form of dystrophin to muscle cells known as Elevidys micro-dystrophin. It is a single-dose gene transfer therapy.

Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of Elevidys in patients with Duchenne muscular dystrophy who are non-ambulatory.

As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to develop and commercialize Elevidys. Sarepta is responsible for regulatory approval and commercialization of Elevidys in the United States, as well as manufacturing. Roche is responsible for regulatory approvals and bringing Elevidys to patients across the rest of the world.

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