Tag

Elevidys

All articles tagged with #elevidys

health1 month ago•14 min saved

ELEVIDYS Slows Duchenne Progression in Three-Year EMBARK Results

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.

via Sarepta Therapeutics|

#duchenne-muscular-dystrophy #elevidys #embark-trial

business2 months ago•1 min saved

2025 Biopharma CEO's Decisions Lead to Tragedy

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.

via statnews.com|

#biopharma #business #ceo

business6 months ago•3 min saved

Sarepta Therapeutics Faces Lawsuit and Investor Investigation

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.

via TipRanks|

#business #elevidys #fda-safety-communication

business7 months ago•1 min saved

Sarepta Stock Surges as FDA Reauthorizes Elevidys Shipments

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.

via Investor's Business Daily|

#biotech #business #drug-shipments

business7 months ago•1 min saved

Sarepta Shares Surge as FDA Approves Resumption of Elevidys Shipments

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.

via Yahoo Finance|

#business #duchenne-muscular-dystrophy #elevidys

health7 months ago•1 min saved

FDA Approves Sarepta's Duchenne Therapy for Younger Patients Amid Ongoing Investigations

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.

via statnews.com|

#duchenne-muscular-dystrophy #elevidys #fda

health7 months ago•1 min saved

FDA Investigates Gene Therapy Deaths Amid Calls for Resumption

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.

via fda.gov|

#duchenne-muscular-dystrophy #elevidys #fda

healthcare7 months ago•7 min saved

FDA Reviews Safety of Sarepta's Elevidys Amid Ongoing Concerns

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.

via Sarepta Therapeutics|

#duchenne-muscular-dystrophy #elevidys #fda-investigation

health7 months ago•1 min saved

EU Rejections and Setbacks for Duchenne Muscular Dystrophy Gene Therapies

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.

via statnews.com|

#duchenne-muscular-dystrophy #elevidys #europe

business7 months ago•3 min saved

Sarepta Faces FDA Scrutiny and Shipment Halt Amid Gene Therapy Concerns

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.

via Investor's Business Daily|

#business #elevidys #fda

health7 months ago•2 min saved

Sarepta Therapeutics Suspends ELEVIDYS Shipments Amid FDA Dispute

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.

via statnews.com|

#duchenne-muscular-dystrophy #elevidys #families

health7 months ago•1 min saved

Sarepta Therapeutics Faces Regulatory and Market Challenges Amid Shipment Pauses and Stock Decline

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.

via statnews.com|

#duchenne-muscular-dystrophy #elevidys #fda

business7 months ago•3 min saved

Sarepta Pauses Elevidys Shipments Amid Safety Concerns

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.

via Investor's Business Daily|

#business #elevidys #fda

business7 months ago•0 min saved

Sarepta Pauses U.S. Elevidys Shipments Amid FDA Concerns

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.

via CNBC|

#business #duchenne-muscular-dystrophy #elevidys

health7 months ago•1 min saved

Sarepta Therapeutics Pauses Duchenne Gene Therapy Shipments Amid Safety Concerns

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.

via statnews.com|

#duchenne-muscular-dystrophy #elevidys #fda