Sarepta (SRPT) gets FDA approval on new gene therapy study
Sarepta Therapeutics (SRPT) announced on Tuesday that it has gotten approval from the Food and Drug Administration (FDA) for an enhanced immunosuppressive regimen that is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) for patients undergoing AAV gene therapy.
The biotech company will be testing a dosing regimen as part of a study cohort for its Elevidys gene therapy that's administered to patients with Duchenne muscular dystrophy.
This dosing regimen involves testing to see whether administering sirolimus prior to and after Elevidys can reduce ALI, which is a known risk associated with AAV gene therapy.
Sarepta will enroll 25 non-ambulatory patients in the US as part of its cohort study.
The company said that the immunosuppressive regimen will include 14 days of peri-infusion sirolimus dosing prior to administering Elevidys and then will included 12 weeks of evaluation after the gene therapy administration.
Sarepta's stock rose 2.3% on news of the FDA approval.
“We remain deeply committed to serving all individuals living with Duchenne, including those who have lost the ability to walk," Dr. Louise Rodino-Klapac, president of research & development and technical operations for Sarepta, said in a statement.
"Guided by real-world experience, external clinician expertise, and FDA input, Cohort 8 of the ENDEAVOR study will evaluate integrating sirolimus into our immunosuppression approach, with the goals of mitigating the risk of acute liver injury and restoring access for non-ambulant individuals living with Duchenne."
Duchenne muscular dystrophy is a rare genetic disorder that’s mostly found in boys and gradually weakens their muscles. Few of those diagnosed with it live past their 20s.
Sarepta's stock has plummeted during a volatile year
Sarepta has faced some significant struggles and controversies with its Elevidys gene therapy this year.
After a 16-year-old boy who had been administered Elevidys died of acute liver failure, the FDA asked Sarepta to suspend shipments of Elevidys and halted all clinical trials using the same gene therapy platform.
The 16-year-old boy was the third patient to die after being administered the gene therapy.
However, after the FDA issued its warning, Sarepta shockingly stood its ground and refused to comply with the regulator.
Sarepta said it had seen “no new or changed safety signals,” and had no plans to stop shipping. The company's refusal came after the drug generated $282 million in Q2 sales, more than half of Sarepta’s total revenue for the quarter.
In a statement at the time, the company said it “will continue to ship ELEVIDYS to the ambulant population” and looks forward to “continued discussions” with the FDA.
Despite its seeming refusal to do so, Sarepta did end up briefly pausing shipments of its gene therapy. But then the FDA backed off after some patient families and right-wing activists close to President Trump pushed back against the regulator's actions, as the Associated Press reported.
Sarepta said that a decision on whether to resume commercial dosing for the patients in its study will be made in collaboration with the FDA after reviewing the results.
The company's stock has plunged 84.1% for the year.