Gene therapy maker Sarepta tells FDA it won’t halt shipments despite patient deaths
WASHINGTON (AP) – Drug producer Sarepta Therapeutics (SRPT) said it would not comply with the request of stopping all shipments from the Food and Pharmaceutical Administration on Friday. Gene therapy after the death of a third patient Taking one of the treatments for muscle dystrophy.
The extremely extraordinary movement is the latest movement in a series of events that have attracted the company’s shares for weeks and forced 500 employees to quit. Company FDA It also provides the future usability of the leading therapy called Elevidys.
FDA said on Friday night that the authorities came together with Sarepta and asked him to suspend all sales, but the company said that he rejected it ”. The agency has the authority to withdraw drugs from the market, but the cumbersome regulatory process may take months or even years. Instead, the agency usually makes an informal request and companies almost always sleep.
“We believe in accessing drugs for unrequited medical needs, but we are not afraid to take action immediately when a serious security signal emerges,” FDA commissioner Marty Makary said.
Elevidys is the first gene therapy for Duchenne’s muscle dystrophy in the United States, the fatal muscle intermittent disease affecting men, but has been faced with a study since the clarity of 2023. One -time treatment has received accelerated approval against the recommendations of some FDA scientists who double the effectiveness.
FDA gave full approval last year and expanded the use of treatment to patients 4 years and older, including those who no longer walk. Previously, it was still available for young patients who were still walking.
Sarepta said on Friday that scientific examination shows “new or changing security signals için for young patients with Duchenne, previous stages of the disease. The company said it plans to keep the drug available for these patients.
“We are looking forward to the sharing of constant discussions and information with FDA,” the company said in a statement.
Sarepta stopped the shipments of treatment last month for older men from Duchenne, who gradually destroyed muscle and skeletal power and resulted in early death. The movement watched the deaths of two young children who took the therapy.
The company also confirmed a third death on Friday: a 51 -year -old patient who receives an experimental gene therapy for a different muscle dystrophy. Sarepta said he reported death to FDA on June 20. FDA said on Friday, he said he was waiting for this hearing.
Sarepta stated that the gene therapy in the incident uses a different dose and produced using a different process from Elevids ”.
