Sarepta Therapeutics (SRPT) has received expanded approval from the Food and Drug Administration (FDA) for its Duchenne muscular dystrophy (DMD) drug, Elevidys (delandistrogene moxeparvovec). This decision significantly broadens the eligible patient population to include both ambulatory and non-ambulatory individuals with DMD.
The approval marks a pivotal moment for patients seeking treatment for this debilitating genetic disorder. Elevidys is a gene therapy designed to deliver a functional dystrophin gene to muscle cells, addressing the underlying cause of DMD, which is characterized by a deficiency in the dystrophin protein.
The FDA's decision was met with enthusiasm from the DMD community and investors alike, as evidenced by the surge in Sarepta's stock price following the announcement. This expanded approval underscores the potential of gene therapies to transform the treatment landscape for rare genetic diseases. The therapy is administered as a single intravenous infusion.
DMD is a progressive neuromuscular disease primarily affecting males, with an estimated incidence of 1 in 3,500 to 5,000 male births worldwide. The lack of dystrophin leads to muscle degeneration and weakness, eventually affecting respiratory and cardiac function. Prior to this approval, treatment options were limited, primarily focusing on symptom management and supportive care.
Elevidys' initial approval was under accelerated approval based on a surrogate endpoint. The expanded approval may be contingent on further clinical trials to verify the therapy's long-term clinical benefit. The company is continuing to evaluate the safety and efficacy of Elevidys in ongoing clinical trials to support its long-term use in DMD patients.
