The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
A third death linked to Sarepta Therapeutics’ gene therapy platform reported Thursday may be the final straw for the FDA, which will request that Sarepta voluntarily stop all shipments of its Duchenne muscular dystrophy treatment Elevidys, according to Reuters, which quoted a source familiar with the matter.
In an email to BioSpace, a Health and Human Services representative confirmed the Reuters report. Additionally, an HHS official told BioSpace in the same email, “We are taking a hard look at pulling it from the market.”
In another report, FDA Commissioner Marty Makary told Bloomberg News that the regulator is considering whether Elevidys, an adeno-associated virus (AAV) vector–based gene therapy that was first approved in June 2023, should stay on the market. Sarepta told CNBC, however, that it had not heard from the FDA.
Shares of the biotech were down nearly 37% at the time of publication.
This latest news follows a difficult season for Sarepta, which previously reported the deaths of two teenage patients taking Elevidys from acute liver failure. On Wednesday, Sarepta added a boxed warning to Elevidys’ for acute liver injury and acute liver failure. On Thursday, news broke of a third death, that of a 51-year-old patient in a Phase I trial for Sarepta’s limb-girdle muscular dystrophy (LGMD) gene therapy SRP-9004, which uses the same underlying platform technology as Elevidys. Like the two teenage boys, the man also died of acute liver failure.
It’s been a rollercoaster of a week for Sarepta, which on Wednesday saw its stock leap 18% after investors responded with cautious optimism to a business overhaul that saw the layoffs of around 500 employees and a pivot from gene therapy to its siRNA platform assets.
“The only risk we see moving forward is the death of a third Elevidys patient,” BMO said in a note to investors at the time.
While the third death wasn’t associated with Elevidys, Sarepta’s lack of disclosure earlier this week didn’t sit well with investors. “We think the LGMD patient death could amplify patient hesitancy to use commercial Elevidys and increase investor distrust since the company did not disclose the event on its call,” William Blair wrote in a note to investors early Friday morning.
In a note to investors Friday afternoon, BMO said Sarepta revealed during a management call that the disclosure of this death was not made during Wednesday’s call because given the discontinuation of the SRP-9004 program.
