All articles tagged with #sarepta therapeutics
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.
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.
Dr. Vinay Prasad, a prominent FDA official known for advocating rigorous drug approval and criticizing industry overreach, resigned amid a smear campaign by political and industry allies, highlighting ongoing conflicts between public health interests and industry influence.
Vinay Prasad, a top FDA regulator, resigned amid political controversy over his handling of Sarepta's gene therapy for Duchenne muscular dystrophy, which included halting and then reinstating the treatment following safety concerns. His departure was seen as a positive development for the biotech sector, potentially leading to a more permissive regulatory environment.
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.
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.
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 has unexpectedly decided to pause all shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, following a request from the FDA, reversing its initial rejection of the agency's directive.
Sarepta Therapeutics' stock plummeted after the FDA demanded the halt of shipments of its gene therapy Elevidys due to safety concerns, following the death of a third patient treated with a related gene therapy. The company refused to withdraw Elevidys, citing safety data, but faced severe criticism and a significant drop in stock value, raising questions about its decision-making and credibility amid safety issues in its gene therapy pipeline.
Sarepta Therapeutics' stock plummeted after a third patient died from acute liver failure following gene therapy treatment, raising safety concerns and impacting investor confidence amid ongoing restructuring and regulatory scrutiny.
Sarepta Therapeutics has laid off over one-third of its workforce, about 500 employees, as part of a major cost-cutting strategy following setbacks in its gene therapy for Duchenne muscular dystrophy. The company is also pausing work on several other gene therapies to save approximately $400 million annually, while promoting top executives with salary increases. These measures aim to improve cash flow and maintain financial stability.
Sarepta Therapeutics announced a strategic restructuring to focus on high-impact programs, including a workforce reduction and cost savings of approximately $400 million annually, while updating on ELEVIDYS' label and safety measures. The company aims for long-term growth by prioritizing its siRNA platform and maintaining its Duchenne muscular dystrophy portfolio, with key executive appointments and financial highlights for Q2 2025.
Sarepta Therapeutics' stock has plummeted nearly 86% this year following the FDA's investigation into the deaths of two teenagers who received its gene therapy, Elevidys, for Duchenne muscular dystrophy, raising concerns about the therapy's safety and potential regulatory actions.
Sarepta Therapeutics has paused its gene therapy trial after the deaths of two patients from acute liver failure, raising concerns about the safety of Elevidys, a treatment for Duchenne muscular dystrophy, which was approved under accelerated pathways despite safety warnings.