FDA broadens approval for Sarepta's Duchenne muscular dystrophy gene therapy Elevidys

The US Food and Drug Administration (FDA) has recently expanded the approval of Sarepta Therapeutics' gene therapy, Elevidys (delandistrogene moxeparvovec-rokl), for the treatment of Duchenne muscular dystrophy (DMD). This approval now includes patients aged four years and older who have a confirmed mutation in the DMD gene.

Originally, Elevidys received accelerated approval from the FDA in June of last year, specifically for ambulatory patients aged between four and five years. The new decision extends traditional approval to encompass ambulatory patients aged four years and older and provides accelerated approval for non-ambulatory patients within the same age group.

Duchenne muscular dystrophy is a severe genetic disorder affecting approximately one in every 3,500 male births globally. It is caused by mutations in the gene responsible for producing dystrophin, a protein essential for muscle strength and protection. Typically, symptoms of DMD appear in early childhood and progressively worsen, severely impairing patients' mobility and affecting their heart and respiratory muscles.

Sarepta's gene therapy aims to address the root cause of DMD by introducing a gene into the patient's body that produces Elevidys micro-dystrophin. This protein is a truncated version of normal dystrophin, containing critical domains necessary for muscle function. The therapy is administered as a single intravenous dose.

The FDA's recent approval was based on comprehensive data from multiple studies. These included two double-blind, placebo-controlled trials and two open-label studies, collectively involving 218 male patients with confirmed mutations in the DMD gene.

Doug Ingram, the president and CEO of Sarepta, described this approval as a pivotal moment for the DMD community. Echoing this sentiment, Jerry Mendell, a co-inventor of Elevidys and senior advisor at Sarepta, highlighted the importance of this development. He emphasized that the expanded indication offers a viable treatment option for the majority of boys and young men living with Duchenne muscular dystrophy. Mendell also pointed out that this expansion underscores the robustness of the scientific evidence and the observable improvements in disease progression demonstrated in the studies.

Sarepta Therapeutics holds the responsibility for regulatory approval, commercialization, and manufacturing of Elevidys within the United States. Beyond the US, Roche manages regulatory approvals and distribution efforts to make the therapy available to patients worldwide.