Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is priced at $3.2 million per dose, produced $821 million in net product revenue for the company in 2024.
Sarepta Therapeutics has reported positive results from part two of the EMBARK study, demonstrating the efficacy and safety of the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in improving functional outcomes for people with Duchenne muscular dystrophy (DMD) who are ambulatoryThe data, shared in Jan.27, 2025, news release, showed that treatment with Elevidys led to significant improvements in muscle function and strength, as well as a reduction in the rate of disease progression.
Approved in June of 2023, Elevidys is indicated for the treatment of DMD in ambulatory individuals at least four years of age who have a confirmed mutation in the DMD gene. In June of 2024, Sarepta received accelerated approval for a label expansion in nonambulatory patients four years of age and older with a confirmed mutation in the DMD gene.
DMD is a progressive myopathic disorder that primarily affects young boys, eventually leading to death. Elevidys aims to address the underlying genetic cause of the disease by delivering a functional microdystrophin-encoding gene to muscle cells via a viral vector.
Sarepta is no stranger to the therapeutic space. Their portfolio also includes exon-skipping RNA therapies approved for dystrophin mutations in DMD. Exondys 51, Vyondys 53, and Amondys 45 are antisense oligonucleotides targeting specific DMD genotypes.
Doug Ingram, J.D.
Boston-based company has announced that it will report its fourth quarter and full-year 2024 financial results on Feb. 26. According to a slide deck posted on the company’s investor relations page, Doug Ingram, J.D., the company’s president and CEO, shared shared some of those financial results already at the J.P. Morgan Healthcare Conference in San Francisco. Figures on the slide deck show Sarepta has having $1.7 billion in net product revenue in 2024, almost half of which — $821 million — came from Elevidys. Elevidys is priced at $3.2 million per dose.
Elevidys is the first gene therapy for DMD and appears to be a significant improvement on Sarepta’s existing therapies. Elevidys is intended to be a single treatment and does not need to match a single specific genotype.
The EMBARK study is a multinational, phase 3, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the efficacy and safety of Elevidys in individuals with Duchenne muscular dystrophy (DMD) aged four to seven years.
The study aimed to assess Elevidys's functional benefits in treating DMD, with a focus on the North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR) function tests, and to monitor the treatment's safety profile. The study is structured in two parts; Part one was published last October in Nature.
In EMBARK part two, patients who received a placebo in part one received Elevidys, whereas those treated with Elevidys part one received a placebo.
Crossover-treated patients (those who received a placebo in part one and Elevidys in part two) showed significant functional improvements in the North Star Ambulatory Assessment (NSAA), Time to Rise (TTR), and 10-meter walk/run (10MWR) compared to a well-matched external control group.
Specifically, crossover-treated patients improved by 2.34 points on the NSAA, reduced TTR by 2.70 seconds, and reduced 10MWR by 1.07 seconds.).
Patients treated in part one showed sustained expression of Elevidys micro-dystrophin at week 64 and continued functional improvements at two years post-treatment. The differences in NSAA, TTR, and 10MWR between these patients and the external control group increased from year one to year two, indicating ongoing divergence from the natural progression of DMD.
Furthermore, two-year MRI results for part one treated patients indicated minimal progression in muscle pathology, consistent with the observed functional benefits.
The safety profile of Elevidys remained consistent with previous findings, with no new safety signals observed.
These findings suggest Elevidys provides sustained functional benefits and disease stabilization in ambulatory individuals with DMD with a manageable safety profile. Sarepta intends to conduct a five-year open-label follow-up study.
