FDA Allows Sarepta to Resume Shipping Elevidys Gene Therapy for Duchenne Muscular Dystrophy with Safety Restrictions
FDA has authorized Sarepta Therapeutics to resume shipping ELEVIDYS, a groundbreaking gene therapy for Duchenne muscular dystrophy (DMD), with new safety restrictions in place. ELEVIDYS is a one-time intravenous gene therapy designed to treat both ambulatory and non-ambulatory patients aged 4 and older by targeting the underlying genetic cause of DMD through micro-dystrophin expression in skeletal, respiratory, and cardiac muscles. This treatment offers hope by stabilizing or improving muscle function and potentially slowing disease progression. However, safety monitoring is critical due to risks of immune-mediated myositis and myocarditis, requiring close cardiac and muscle symptom surveillance after administration. ELEVIDYS represents a major advancement in DMD therapy, now approved in multiple countries and expanding treatment options for patients who previously had limited disease-modifying interventions. Early diagnosis remains important to maximize treatment benefits and preserve quality of life.