The swift FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety study, as had been feared, Jefferies analysts said Monday.
The FDA is recommending that the voluntary hold on Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy be lifted for ambulatory patients, barely one business day after announcing an investigation into the death of an 8-year-old Brazilian patient.
In a quick response to the news, Jefferies said the FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety study. The news “significantly improves Elevidys’ sales outlook in the near-term and creates better visibility around Sarepta’s go-forward cost structure.”
Jefferies had been concerned that the FDA might remove Elevidys from the market in ambulatory DMD, even with a full approval. Ambulatory patients represent half of Sarepta’s market for the gene therapy, so the loss of that indication would have been a significant blow.
The regulatory whiplash is the latest twist in the tale of Sarepta’s gene therapy, which began earlier this year as severe safety issues and deaths emerged. After a third patient—a 51-year-old man who had been participating in a Phase I study for Sarepta’s SRP-9004 in limb-girdle muscular dystrophy, which also uses an AAV vector—died, the FDA stepped in, asking the company to suspend all U.S. deliveries of the therapy for ambulatory patients. Then on Friday, another death was revealed, and the FDA began an investigation.
But in a brief statement Monday afternoon, the FDA recommended the removal of the voluntary hold, clearing the way for ambulatory patients to continue to receive Elevidys. The agency agreed with Sarepta and Brazilian authorities that the most recent death of the eight-year-old Brazilian patient was not related to the gene therapy product itself, but likely due to a case of serious influenza A infection intensified by immunosuppression.
At this time, the FDA is not making any recommendations for non-ambulatory patients, for which a voluntary hold is still in place after two deaths that were linked to Elevidys.
The FDA did not provide any detailed information about its investigation, but said, “the patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD.”
The statement announcing the investigation on Friday was also brief.
The first death linked to Elevidys was reported in March, with a second following in June. Both were tied to acute liver failure, a known complication of the AAV vectors used to deliver gene therapies. Sarepta added a black box warning on the product to address the risk. But then the third death in the limb-girdle muscular dystrophy was revealed earlier this month. The FDA put a hold on all studies in the indication. William Blair analysts said at the time that SRP-9004 uses the same capsid as Elevidys.
Then the fourth death of the Brazilian boy was announced last week, putting Sarepta on its back foot. The biotech and the FDA appeared to butt heads over keeping Elevidys on the market, with the biotech initially refusing to acquiesce to the regulator’s request to pause shipments. The FDA also pulled Sarepta’s platform designation, which had been awarded for the biotech’s viral vector technology.
