FDA Approves Elevidys, the First Gene Therapy for Duchenne Muscular Dystrophy
Groundbreaking Treatment Granted Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Sarepta's gene therapy, delandistrogene moxeparvovec (Elevidys), for the treatment of Duchenne muscular dystrophy (DMD), a rare and debilitating genetic disorder.
About Duchenne Muscular Dystrophy
DMD is an X-linked muscle-wasting disease that primarily affects males. It is characterized by progressive muscle weakness and degeneration, leading to severe disability and a shortened life expectancy.
Elevidys Gene Therapy
Elevidys is a gene transfer therapy that uses an adeno-associated virus (AAV) vector to deliver a functional copy of the dystrophin gene to DMD patients' cells. Dystrophin is a protein that plays a crucial role in muscle function.
Clinical Trial Results
In a Phase 3 clinical trial involving 102 patients, Elevidys was shown to significantly improve muscle function in participants. The therapy led to increases in timed function tests and reductions in disease severity.
Approval and Availability
The FDA's accelerated approval of Elevidys is based on clinical trial data showing the potential for long-term benefit. The therapy is currently available for patients with DMD who are ambulant and have a confirmed mutation in the dystrophin gene.
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