Latest news with #liver failure
Yahoo
4 days ago
- Business
- Yahoo
Third Fatal Liver Failure Hits Sarepta Gene Trial
Sarepta (NASDAQ:SRPT) has now seen a third fatal incident in its gene therapy trials due to acute liver failure. That happened last month in a limb girdle muscular dystrophy study and follows two teenage deaths in advanced Duchenne muscular dystrophy patients. The trial was halted and Elevidys distribution paused for non walking patients. Warning! GuruFocus has detected 3 Warning Signs with SRPT. The company reported the latest death promptly to regulators and agreed to alert doctors and patients about liver failure risks as the FDA requested. Talks are underway on how to protect non walking patients going forward. To steady its finances, Sarepta will cut more than a third of its workforce and pause several drug programs. This move is expected to save around four hundred million dollars a year. Investors will watch closely for signs that these steps can rebuild confidence. This article first appeared on GuruFocus.
New York Times
4 days ago
- Health
- New York Times
F.D.A. to Ask Biotech Company to Stop Shipping Treatment Linked to Patient Deaths
The Food and Drug Administration will request that the biotech company Sarepta Therapeutics halt all shipments of its treatment for a deadly muscle-wasting disease after three patients died from liver failure after taking the product or a similar therapy. The troubled treatment, known as Elevidys, has fueled an ongoing debate over whether the F.D.A. has grown too lenient in approving drugs that are insufficiently safe or effective. New officials in the Trump administration, however, have rejected several new drug applications and narrowed the use of Covid vaccines over concerns about 'unknown' side effects. Elevidys, a gene therapy, is administered as a one-time intravenous infusion. It is intended to slow the progression of Duchenne muscular dystrophy, which causes muscles to deteriorate. The disease often kills patients, typically young men, before their 30th birthday. Elevidys, first approved in 2023, is one of a handful of treatments for the disease that arrived in the past decade, with the help of fierce advocacy from patients and their families. But the approval of Elevidys was controversial because of limited evidence that its benefits outweighed its risks. In recent months, two teenage boys who had received Elevidys died of liver failure, deepening critics' concerns about the drug and prompting speculation that the F.D.A. would take strict action. In late June, the F.D.A. announced an investigation into the deaths. Liver injuries were among the side effects seen in clinical trials of the treatment. On Tuesday, the company said it would halt shipments for patients with more advanced disease who use wheelchairs, a group that includes the teenagers who died and most Duchenne patients over age 12. At the time, the company planned to keep distributing the product for patients who could still walk. Want all of The Times? Subscribe.
Reuters
4 days ago
- Business
- Reuters
Sarepta shares slump after third patient death this year
July 18 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab declined 17% in early trading on Friday after a patient who had received one of the company's experimental gene therapies died, deepening investor concern over the use of its treatments. Sarepta said a 51-year-old man enrolled in an early-stage trial of gene therapy, SRP-9004, for the treatment of limb-girdle muscular dystrophy (LGMD) died from acute liver failure last month. This is the third death this year for Sarepta after two teenage boys who had received Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy, died of liver failure. Sarepta said the patient was the last to be dosed in that study. LGMD includes a group of genetic disorders that primarily weaken the hip and shoulder muscles. The news of the death was first reported by pharma website BioCentury on Thursday evening. On Wednesday, Sarepta announced a restructuring plan that included 500 job cuts and halting development of several gene therapies for limb-girdle muscular dystrophy. It said then the cuts were a financial decision and that it hopes to find partners for these programs. The company said it plans to host a call with investors later on Friday.
Reuters
4 days ago
- Business
- Reuters
Sarepta shares slump on reports of third patient death this year
July 18 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab declined 20% in early trading on Friday after media reports that a patient who had received one of its experimental gene therapies has died, deepening investor concern over the use of the company's treatments. This is the third death this year for Sarepta after two teenage boys who had received Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy, died of liver failure. A 51-year-old man enrolled in an early-stage trial of Sarepta's gene therapy, SRP-9004, for the treatment of limb-girdle muscular dystrophy (LGMD) died from acute liver failure last month, according to several media reports. LGMD includes a group of genetic disorders that primarily weaken the hip and shoulder muscles. The news of the death was first reported by pharma website BioCentury on Thursday evening. On Wednesday, Sarepta announced a restructuring plan that included 500 job cuts and halting development of several gene therapies for limb-girdle muscular dystrophy.
Reuters
4 days ago
- Business
- Reuters
Sarepta shares slump after report of third patient death this year
July 18 (Reuters) - Shares of Sarepta Therapeutics (SRPT.O), opens new tab declined 16.5% in premarket trading on Friday after a Bloomberg report that a trial patient who received an experimental gene therapy has died from liver failure, making it the third such fatality this year. The patient, who died last month, was enrolled in an early-stage study testing Sarepta's gene therapy to treat a muscle-wasting disorder called limb-girdle muscular dystrophy, the report said, citing a company spokesperson. Two teenage boys died earlier this year from liver toxicities after they received Sarepta's Elevidys, a gene therapy approved to treat a rare condition called Duchenne muscular dystrophy. Both patients were non-ambulatory, i.e., they were unable to walk independently. The fatalities have brought Sarepta under heightened regulatory scrutiny, and cast doubts on the safety and future demand for Elevidys. Sarepta did not immediately respond to a Reuters request for comment outside of its business hours. It has informed regulatory authorities and the trial investigators about the patient's death, according to the report. The company said on Wednesday it had halted development of several experimental gene therapies for limb-girdle muscular dystrophy. It has also said it would cut 500 jobs, and add a serious warning on the label of Elevidys for the risk of acute liver injury and liver failure in patients with Duchenne muscular dystrophy who can walk. The U.S. Food and Drug Administration gave traditional approval to Elevidys for patients aged four and above who can walk, and conditional approval for those who cannot, in 2024 despite the therapy failing to meet the main goal in a late-stage study.
