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Sarepta Therapeutics Expands FDA Approval of ELEVIDYS to Include Duchenne Muscular Dystrophy Patients Ages 4 and Above

NextEdge Admin

21 Jun 2024

3 min 7 sec

Sarepta Therapeutics, Inc. has achieved a significant milestone with the expansion of the FDA-approved indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include Duchenne muscular dystrophy (DMD) patients aged 4 and above, irrespective of ambulatory status. This decision follows extensive clinical trials and marks a pivotal advancement in precision genetic medicine for rare diseases.

Doug Ingram, President and CEO of Sarepta, emphasized the monumental impact of this approval on the Duchenne community, hailing it as a triumph for gene therapy and scientific advancement. He credited Drs. Jerry Mendell and Louise Rodino-Klapac for their relentless dedication spanning two decades in developing this therapy. Ingram expressed gratitude to the FDA for their evidence-based approach, expediting access to this life-saving treatment for patients in need, and acknowledged the contribution of clinical investigators and courageous Duchenne families.

Dr. Jerry Mendell, co-inventor of ELEVIDYS and senior advisor at Sarepta, highlighted the culmination of his 50-year quest to find effective treatments for Duchenne patients. He underscored the expanded label's significance, noting it provides clinicians with a crucial therapeutic option for the majority of individuals affected by Duchenne. Dr. Mendell also recognized the scientific progress and improvements observed in disease management through extensive study outcomes.

In adherence to the accelerated approval pathway, Sarepta committed to conducting a randomized, controlled trial (ENVISION) to confirm ELEVIDYS's clinical benefits in non-ambulatory Duchenne patients. This Phase 3 study aims to fulfill postmarketing requirements, ensuring continued assessment of treatment efficacy.

Moreover, Sarepta's collaboration with Roche, initiated in 2019, aims to enhance the global availability of ELEVIDYS, with Sarepta handling U.S. regulatory approvals, commercialization, and manufacturing, while Roche focuses on international regulatory pathways and global distribution.

The expanded indication for ELEVIDYS represents a culmination of scientific innovation and dedication, promising hope for those affected by Duchenne muscular dystrophy worldwide. This approval underscores Sarepta's commitment to advancing precision genetic therapies and transforming outcomes for patients with rare genetic diseases.