Cambridge, MA July 22, 2025:
Sarepta Therapeutics, a leader in precision genetic medicine for rare diseases, has voluntarily paused all U.S. shipments of ELEVIDYS (delandistrogene moxeparvovec) a groundbreaking gene therapy for Duchenne muscular dystrophy (DMD) effective immediately. The company notified the U.S. Food and Drug Administration (FDA) regarding the decision, which was taken as part of an ongoing collaboration to finalize supplemental safety labeling.
This proactive pause reflects Sarepta’s unwavering commitment to patient safety and transparency. The company will utilize this time to address FDA’s data requests and ensure that ELEVIDYS continues to meet the highest standards of care in the treatment of Duchenne muscular dystrophy.
ELEVIDYS: A Pioneering Gene Therapy for DMD
ELEVIDYS is a one-time intravenous infusion gene therapy, designed to treat DMD in 4-year-old patients. It works by delivering a functional transgene encoding ELEVIDYS micro-dystrophin, targeting the underlying cause of Duchenne mutations in the DMD gene that lead to insufficient production of dystrophin protein in muscles.
Despite its promise, clinical data revealed certain safety concerns, including:
Acute serious liver injury, often within 8 weeks post-infusion, with elevated biomarkers such as ALT, AST, GGT, GLDH, and total bilirubin.
Immune-mediated myositis, observed in patients with deletions involving exon 8 and/or exon 9 of the DMD gene, around a month after dosing.
For more detailed prescribing and safety information, patients and healthcare providers are encouraged to visit FDA MedWatch.
CEO’s Statement: A Heartfelt, Patient-First Decision
Doug Ingram, President and CEO of Sarepta Therapeutics, commented:
“As a patient-focused company, this decision was incredibly difficult. We understand that every day matters for individuals with Duchenne. However, it’s vital for us to maintain a positive and collaborative relationship with the FDA, ensuring ELEVIDYS can continue to be a safe and effective treatment. This temporary pause will allow us to align on the safety label updates and provide the clarity FDA seeks.”
He added that the company remains committed to delivering updates as new information becomes available, including guidance for healthcare professionals, patients, and the broader Duchenne community.
What This Means for Patients and the Future of ELEVIDYS
While the pause may delay access temporarily, it sets the stage for long-term sustainability, regulatory trust, and greater treatment clarity. The ELEVIDYS label supplement is expected to enhance confidence among clinicians and patients by clearly outlining potential risks, particularly for certain genetic subtypes of DMD.
As Sarepta moves forward with FDA cooperation, ELEVIDYS remains a beacon of innovation in the battle against Duchenne muscular dystrophy, offering hope for a life-altering therapeutic option in a space with limited disease-modifying treatments.
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