In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The designation, granted last month, was the first of its kind to be announced publicly.
The safety saga over Sarepta Therapeutics’ Duchenne muscular dystrophy drug Elevidys reached tense heights on Friday, when the biotech refused the FDA’s request to stop all shipments of the gene therapy in the U.S. The FDA on Friday also revoked the platform designation for Sarepta’s viral vector technology.
The request, according to the biotech, was an “informal” one, which the company only became aware of “at the same time the public and our patient communities did, through media reports,” according to a Friday news release. “We will continue to ship Elevidys to the ambulant population,” Sarepta said, citing its own “comprehensive scientific interpretation” of the therapy’s safety data.
In its own news release on Friday, the FDA claimed that it had met with Sarepta to request a voluntary suspension of all Elevidys shipments. “The company refused to do so,” according to the regulator. “The FDA will not allow products whose harms are greater than benefits,” Vinay Prasad, director of the Center for Biologics Evaluation and Research, said in the statement.
It remains unclear what action the FDA will take against Sarepta in light of the biotech’s refusal to pause shipments, though the agency on Friday said that it will review Elevidys’ safety data in the ambulatory patient population and “will take additional steps to protect patients as needed.”
Citing recent safety issues, the FDA on Friday also took back its Platform Technology Designation for rAAVrh74, Sarepta’s adeno-associated virus (AAV) vector platform, which it uses to deliver its gene therapies. The award was initially granted last month—the first of its kind to be publicly disclosed—in a move that William Blair analysts at the time said would help Sarepta “accelerate the development of its follow-on gene therapy programs” and lower early R&D spend.
Sarepta reported the first patient death in March, followed by a second mortality in June. Both deaths, linked to Elevidys, were attributed to acute liver failure, a known complication of the AAV vectors used to deliver gene therapies. Last week, the biotech moved to put a black box warning on Elevidys’ label, alongside a slew of corporate restructuring initiatives that sent its stock soaring some 18%.
The rise was short-lived, however, as reports of a third patient death surfaced Friday. This latest mortality was not associated with Elevidys, but with SRP-9004, a gene therapy being tested for limb-girdle muscular dystrophy (LGMD). The FDA on Friday put a clinical hold on all of Sarepta’s LGMD studies. Sarepta did not disclose this third death during its corporate update earlier in the week.
In a statement to BioSpace at the time of the third death, a company spokesperson insisted that Sarepta had already relayed the news of the third death to regulators “in an appropriate and timely manner.” Sarepta has also “transparently disclosed this tragic event to the community with whom we have partnered closely throughout the development process,” the spokesperson said.
In an investor call on Friday, company management stated that the latest patient death was “not material” to investors, which is why it left it out of the corporate update.
In a note to investors on Friday, William Blair analysts disagreed, noting that “SRP-9004 uses the same capsid” as Elevidys. Sarepta’s “disclosure protocols could amplify patient hesitancy to use commercial Elevidys and increase investor distrust,” William Blair added, contending that “the lack of transparency” is contributing to the drop in Sarepta stocks.
