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 reported a third patient death due to acute liver failure after receiving its gene therapy, adding to recent fatalities of two teenage boys, which has led to stock decline and increased regulatory scrutiny, including a warning label from the FDA.
Sarepta's stock plummeted after the death of a second patient treated with its Duchenne muscular dystrophy gene therapy, Elevidys, leading to a pause in clinical trials and shipment suspensions, with analysts lowering sales expectations and raising concerns about regulatory and safety implications.
Sarepta Therapeutics has halted shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, after the death of a second non-ambulatory patient due to acute liver failure, prompting the company to work with experts and the FDA to develop a safer immunosuppressive regimen.
A patient died following an experimental gene therapy trial by Rocket Pharmaceuticals for Danon disease, prompting the FDA to halt the study and investigate the cause, with concerns centered around a novel immune suppression agent used in the treatment regimen.
A participant in Rocket Pharmaceuticals' gene therapy trial for Danon disease died, with the company investigating the cause, possibly related to immune suppression drugs used before therapy.
An 81-year-old patient died at CHI Saint Joseph Health Main in Lexington due to a medication mix-up, according to a coroner's report. The patient was mistakenly given Naturalyte, a dialysis liquid, instead of a colonoscopy prep medication. The nursing board's investigation revealed multiple process failures, including a barcode scanning issue and a workaround that led to the administration of the wrong medication. The nurse involved was not disciplined by the nursing board, but concerns were raised about not visually double-checking the medication's label. The incident has prompted discussions about the complexities of healthcare, the over-reliance on technology, and the need for human vigilance in preventing medication errors. The Kentucky Board of Pharmacy is currently investigating the incident.
Dr. Victoria Mondloch, a doctor from Waukesha, has permanently surrendered her medical license following the death of a patient from late-stage endometrial cancer. The Wisconsin Medical Examining Board suspended her license after she performed a biopsy on the patient in violation of previous board action and provided experimental treatments. This is not the first time Mondloch has faced disciplinary action, as she has been reprimanded in the past for incidents including delivering twins vaginally despite signs of distress and cutting a baby's head during delivery.
Arcellx's CAR-modified T cell therapy trial for multiple myeloma has been put on hold after a patient died. The therapy is the company's lead program and part of Gilead's efforts to commercialize additional cell therapies. Gilead contributed $225 million upfront on the program last year.
Seattle Children's has paused a Phase I study of 2seventy bio's CAR-T cell therapy, SC-DARIC33, following the death of a patient who received the therapy at the second dose level. The therapy is being investigated in pediatric and young adult patients with acute myeloid leukemia, and the cause of the adverse event is currently under investigation.