WASHINGTON (AP) – Drug maker Sareepta Therapeutics (SRPT) said at the end of Friday that it will not meet a request from the Food and Drug Administration to stop all shipments of his gene therapy after the death of a third patient who received one of his treatments for muscular syrophy.
The very unusual move is a last in a series of events that the shares of the company have been haaming for weeks and have recently forced it to dismiss 500 employees. The decision of the company not to comply with the FDA also question the future availability of its leading therapy, called Elevidys.
The FDA said on Friday evening in a statement that civil servants met Sareepta and asked to suspend all sales, but “the company refused to do this.” The agency has the authority to remove medication from the market, but the cumbersome regulation process can take months or even years. Instead, the agency usually makes an informal request and companies almost always satisfy.
“We believe in access to drugs for unfulfilled medical needs, but are not afraid to take immediate action when a serious safety signal arises,” said FDA commissioner Marty Makary in a statement.
Elevidys is the first gene therapy approved in the US for the muscular dystrophy of Duchenne, the fatal muscle -wasting disease that affects men, although it has had to do since the approval in 2023. The one-off treatment received an accelerated approval against the recommendations of some FDA scientists who have doubted its effectiveness.
The FDA gave full approval last year and expanded the use of the therapy of 4 years and older, including those who can no longer walk. Previously it was only available for younger patients who were still walking.
Sareepta said on Friday that his scientific assessment “no new or changed safety signals” demonstrated for younger patients with Duchenne's who have previous stages of the disease. The company said it is planning to keep the medicine available to those patients.
“We look forward to constant discussions and sharing information with the FDA,” the company said in a statement.
Sarepta stopped the shipments of therapy last month for older boys with those of Duchenne, who gradually destroys muscle and skeletal strength, resulting in early death. The move followed the death of two teenage boys who took the therapy.
The company also confirmed a third death Friday: a 51-year-old patient who took an experimental gene therapy in a study for a different form of muscular dystrophy. Sareepta said it reported death to the FDA on 20 June. The FDA said on Friday that it had put that process on hold.
Sareepta noted that the gene therapy involved in the incident uses “a different dose and is manufactured with the help of a different process” than Eimidys.
