The Food and Drug Administration (FDA) may revoke authorization for a gene therapy for which multiple deaths have been reported, the agency’s commissioner said on July 18.
The FDA is “taking a hard look” at whether Sarepta Therapeutics’ Elevidys should remain on the market, Dr. Marty Makary told Bloomberg.
Elevidys, a treatment for a rare form of muscular dystrophy called Duchenne muscular dystrophy, was approved after a previous top FDA official, Dr. Peter Marks, overrode recommendations from staffers that determined that clinical data did not show it benefited patients, according to FDA documents.
Dr. Vinay Prasad, who succeeded Marks as head of the FDA’s Center for Biologics Evaluation and Research, has criticized the decision. In a social media post before joining the government, he wrote that Marks “is approving Gene Therapies that don’t work,” while in another post, he said that Marks “overrode 3 reviewers to approve gene therapy that kills more boys [than] it saves.”
Two patients have since died after receiving the gene therapy, the company has disclosed. It has paused shipments of Elevidys for nonambulatory patients for now.
Company officials on July 18 told investors in a call that a third patient died of acute liver failure in a clinical trial after receiving a different gene therapy for limb-girdle muscular dystrophy.
The patient was 51 years old, nonambulatory, and received the gene therapy in a phase 1 trial, according to executives.
They said the death happened a month ago and that they informed the FDA soon after it happened. They defended not disclosing the death in a call on July 16, describing it as unrelated to that call’s focus, which was a restructuring plan that included the halt of multiple trials for gene therapies.
Investors on the call pressed executives about the position.
“It seems clearly that this was a material event,” one said, noting that in the July 16 call, his colleague had specifically asked about the safety of gene therapy for limb-girdle muscular dystrophy.
“We did not discuss this matter in our call on Wednesday because it was neither material nor central to the topics at hand,” Sarepta CEO Douglas Ingram said.
The decision to suspend the trials did not relate to any safety events in the limb-girdle muscular dystrophy trials, Ingram said.
In addition to announcing the discontinuance of trials, executives said in the July 16 call that they had agreed with an FDA request to include a black box warning for acute liver injury and liver failure in the labeling for Elevidys.
The company also stated that it was firing about 500 employees, or about a third of its workforce, as part of cost-cutting measures aimed at saving $400 million on an annual basis.