All articles tagged with #duchenne muscular dystrophy
Sarepta Therapeutics said CEO Doug Ingram will retire by year-end after a decade in which he steered the company to three Duchenne drug approvals and a peak value around $15 billion, followed by a downturn as safety concerns around its gene therapy and the rise of superior rivals weighed on the stock; Ingram cited family health reasons—his wife and son were diagnosed with myotonic dystrophy—and Sarepta is searching for a successor.
Sarepta reports three-year topline results from EMBARK showing ELEVIDYS significantly slows disease progression in ambulatory Duchenne patients treated at ages 4–7. By year 3, NSAA remained above baseline, with a 73% slowing of progression by Time to Rise and a 70% slowing by 10-meter walk/run versus a pre-specified external control; safety signals align with prior data, including a boxed warning for acute liver injury and related risks, with no new safety concerns observed.
FDA has requested the full HOPE-3 clinical study report (CSR) and supporting data to address Capricor’s Complete Response Letter for the Deramiocel BLA; Capricor will submit the CSR and updates in February 2026 to support continued FDA review and a new PDUFA date, following HOPE-3 topline data showing significant skeletal muscle and cardiac improvements in Duchenne muscular dystrophy.
Doug Ingram of Sarepta Therapeutics is criticized as the worst biopharma CEO of 2025 for making risky decisions to push for broad approval of the gene therapy Elevidys for Duchenne muscular dystrophy, despite limited safety and efficacy data, leading to tragic consequences.
U.S. Secretary of Health and Human Services has approved adding Duchenne Muscular Dystrophy and Metachromatic Leukodystrophy to the newborn screening panel, enabling earlier diagnosis and treatment to improve outcomes for affected children.
Sarepta Therapeutics' stock plummeted by over a third after reporting mixed Q3 results, including two drug failures and delayed test outcomes, despite strong sales of its gene therapy Elevidys. The company plans to seek traditional FDA approval for Amondys 45 and Vyondys 53, but uncertainties around regulatory and reimbursement issues persist. Meanwhile, sales of Elevidys exceeded expectations, though the outlook remains uncertain amid regulatory and demand challenges.
Two brothers with Duchenne muscular dystrophy received gene therapy, which significantly improved one brother's condition, but insurance denied coverage for the other, raising concerns about fairness and access to life-changing treatments.
Two brothers with Duchenne muscular dystrophy received gene therapy, which significantly improved one brother's condition, but insurance denied coverage for the other, citing lack of medical necessity, raising ethical and legal concerns for their family.
The FDA is currently facing turmoil after approving Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy in 2023, which was later linked to three patient deaths from liver failure, leading to a suspension request and a sharp decline in Sarepta's stock value.
Sarepta Therapeutics' shares rebounded after the FDA approved the resumption of shipments for its gene therapy Elevidys in the US for ambulatory patients with Duchenne muscular dystrophy, easing concerns about liquidity and market withdrawal, though shipments for non-ambulatory patients remain halted following recent deaths and regulatory scrutiny.
The FDA has allowed Sarepta Therapeutics to resume shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, for younger patients who can walk, after a brief halt due to safety concerns related to non-ambulatory patients following two deaths. The agency continues to review the therapy for non-ambulatory patients, where a voluntary hold remains in effect.
The FDA recommends lifting the voluntary hold on Elevidys for ambulatory patients with Duchenne Muscular Dystrophy after concluding that a recent death was unrelated to the therapy, while ongoing concerns for non-ambulatory patients remain under review.
Sarepta Therapeutics issued a statement clarifying that the recent death of an eight-year-old Duchenne muscular dystrophy patient in Brazil, who received their gene therapy ELEVIDYS, was deemed unrelated to the treatment. The FDA is investigating the case, but Sarepta emphasizes their commitment to patient safety and regulatory transparency, highlighting the safety profile and risks associated with ELEVIDYS, including infusion reactions and liver injury.
European regulators have decided not to approve the Duchenne muscular dystrophy gene therapy Elevidys, adding to global safety concerns and halting its distribution in Japan and Brazil, with ongoing investigations into safety issues.
The halt of Sarepta Therapeutics' gene therapy Elevidys for Duchenne muscular dystrophy due to FDA safety concerns has caused heartbreak and uncertainty for families awaiting treatment, highlighting the emotional toll of regulatory decisions on young patients and their families.