FDA Expands Approval of Elevidys Gene Therapy to Include All Ambulatory and Non-Ambulatory DMD Patients Aged 4 and Older

Jul 01, 2024 21:30 CST Updated 21:30

Sarepta

Developer of Therapies for Rare Neuromuscular Diseases

Roche

Oncology Drug Research, Development, and Manufacturing

On June 20, 2024, the U.S. FDA approved the gene therapy jointly developed by Sarepta Therapeutics and Roche.Elevidys（delandistrogenemoxeparvovec，SRP-9001）Expanded indications to include patients with Duchenne muscular dystrophy (DMD) who are at least 4 years old and have confirmed DMD gene mutations.

Elevidys

The U.S. FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients.。The continued approval for non-ambulatory DMD patients depends on the results of subsequent confirmatory trials.Elevidys is contraindicated in patients with deletions in exon 8 and/or exon 9 of the DMD gene.

ElevidysAiming to address the root cause of DMD by inducing the functional components of dystrophin in muscle tissue; a transgene encoding a truncated form of dystrophin (mini-DMD) is packaged into an AAV viral vector, and a single intravenous injection enables the patient's muscles to produce a recombinant protein with partial dystrophin functionality, effective for patients carrying any type of DMD-causing genetic mutation. There is one distinction from most gene therapies: the oversized DMD gene cannot fit into conventional gene therapy vectors, but a miniaturized version of the gene overcomes this limitation. Despite the absence of the full DMD gene, the core components are still present, allowing it to function almost identically to the complete gene.

On June 22, 2023, Elevidys, the world's first one-time gene therapy for Duchenne Muscular Dystrophy (DMD), was approved by the U.S. FDA for marketing to treat independently walking DMD children aged 4-5.

DMD Duchenne Muscular Dystrophy

In 2014, the international organization United Parent Muscular Dystrophy (UPMD) designated September 7th asDMD World Awareness Day.

DMD, or Duchenne Muscular Dystrophy, is the most common form of muscular dystrophy, where muscles in the body gradually weaken. It is an X-linked recessive muscle disease caused by mutations in the dystrophin gene. The incidence rate in male newborns is approximately 1/3500.

Through genetic testing,93.1% of patients have a genetic cause., laying the foundation for early gene therapy. The causative gene of DMD is the dystrophin gene, located in the Xp21.2 region of the chromosome, with a total length of approximately 2.2Mb, comprising 79 exons, making it the largest known human gene; they are responsible for producing dystrophin, a protein essential for muscle cell health. DMD gene mutations mainly involve exon deletions/duplications, followed by point mutations in the internal exons and flanking regions of the gene. Due to these gene mutations, patients lack dystrophin in their muscle tissue, resulting in muscle weakness and progressive muscle loss.As the condition continuously worsens with age, the full-body muscle weakness affects the heart and breathing as children with the disease grow into teenagers.

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