All articles tagged with #elevidys
FDA plans advisory panels to reconsider compounded peptides; a sweeping Cochrane review revives debates over amyloid-targeting Alzheimer’s drugs; Roche will run another Elevidys trial outside the U.S. as Sarepta markets it domestically; Lilly’s Foundayo GLP-1 pill shows non-inferiority to insulin glargine with a FDA submission planned by the end of Q2.
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.
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.
A class action lawsuit was filed against Sarepta Therapeutics alleging that the company and its officers made false and misleading statements about the safety and efficacy of its gene therapy drug Elevidys for Duchenne muscular dystrophy, omitting critical safety concerns that led to FDA investigations and a significant stock decline.
Sarepta's stock surged after the FDA allowed the company to resume shipments of its gene therapy drug Elevidys for Duchenne muscular dystrophy, following a voluntary pause for safety review. The stock closed up 14.2%, though it has declined significantly this year.
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.
Sarepta's stock dropped after the FDA indicated it will require additional clinical testing for its gene therapy Elevidys, following recent safety concerns and patient deaths, raising concerns about setting a dangerous precedent for gene therapies. The company has already halted sales of Elevidys for certain patients, and the situation continues to create uncertainty for its future and the gene therapy industry.
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.
Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy, Elevidys, faces significant hurdles to return to the market after safety concerns, including liver injuries linked to patient deaths, making its re-approval challenging and potentially risking license revocation, according to a senior FDA official.
Sarepta Therapeutics announced it will voluntarily halt all shipments of its gene therapy Elevidys in the U.S. to address FDA concerns following patient deaths and label updates, leading to a significant stock decline and raising questions about its future prospects and regulatory relationships.
Sarepta Therapeutics announced it will temporarily pause all shipments of its Elevidys gene therapy in the U.S. following the death of a patient who received a different experimental treatment, after initially refusing to halt shipments despite regulatory requests.