FDA Expands Approval of Sarepta Therapeutics’ Elevidys for the Treatment of Duchenne Muscular Dystrophy

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The FDA has expanded its approval of Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec), now indicated to treat patients aged 4 years and older with Duchenne muscular dystrophy (DMD) regardless of their ambulatory status, granted they have a confirmed mutation in the DMD gene. The FDA granted a traditional approval to the drug for the treatment of ambulatory patients, with an accelerated approval for non-ambulatory patients pending results of the Phase III ENVISION study. Elevidys should not be taken by patients with deletions in exon 8 and/or exon 9 of the DMD gene.¹

“Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, president, CEO, Sarepta, in a press release. “At this pivotal moment, I want to give warm thanks to Drs. Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease, to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients, and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.”

The global, randomized, double-blind, placebo-controlled ENVISION trial evaluated non-ambulatory and older ambulatory individuals with DMD. Currently in progress, the study will further support the ongoing approval process for Elevidys. Adverse events (AEs) in clinical studies for Elevidys included vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

Sarepta warns that the use of Elevidys has led to infusion-related reactions, such as hypersensitivity reactions and anaphylaxis up to several hours after treatment. Additionally, acute liver serious liver injury has been observed. Approximately one month after treatment, immune-mediated myositis was also reported in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Further, myocarditis and troponin-I elevations were also found.

As a part of a collaboration with Roche, Sarepta will take the lead on regulatory approval, commercialization, and manufacturing of Elevidys in the United States, while Roche will handle international regulatory approvals and distribution.¹

Regarding current treatment for DMD, steroids have demonstrated improvement in muscle strength and function for patients aged 6 months to 2 years of age, while also slowing down muscle weakening. According to the United Kingdom’s National Health Service (NHS), research suggests that a daily dose proves to be the most effective. NHS also cites ataluren as a treatment for patients ages 2 years old and up who have the ability to walk. Other treatments include creatine supplements and corrective surgery.²

“Today’s expansion of the Elevidys label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle,” said Jerry Mendell, MD, co-inventor of Elevidys, senior advisor, Medical Affairs, Sarepta, in the press release. “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. 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.”

References

1. Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above. Business Wire. June 20, 2024. Accessed June 21, 2024. https://www.businesswire.com/news/home/20240620640445/en/Sarepta-Therapeutics-Announces-Expanded-US-FDA-Approval-of-ELEVIDYS-to-Duchenne-Muscular-Dystrophy-Patients-Ages-4-and-Above 

2. Treatment-Muscular dystrophy/ NHS. Accessed June 21, 2024. https://www.nhs.uk/conditions/muscular-dystrophy/treatment/#:~:text=In%20people%20with%20Duchenne%20MD,the%20process%20of%20muscle%20weakening.