The US FDA has ordered Sarepta Therapeutics to conduct new analyses for its Duchenne muscular dystrophy (DMD) gene therapy Elevidys due to concerns over liver function test results. The company had already paused shipments of the treatment in the US following a series of controversies and deaths linked to the drug.
The FDA wants Sarepta to alter its dosing or manufacturing process for Elevidys and test it on a smaller group of patients to improve liver function test results, rather than conducting a full clinical trial. This approach is seen as more manageable and follows established regulatory precedent.
However, no one at the FDA thinks that Elevidys should return to market based on current data. The company’s focus remains on serving patients, with Sarepta stating it will work within the ordinary and well-established FDA channels and procedures.
Sarepta initially resisted the FDA’s request, but eventually paused shipments of Elevidys in May after reports surfaced of two patients dying after receiving the treatment. Since then, the company has taken steps to update safety labeling and risk management schemes for the gene therapy.
It remains unclear whether the FDA has the authority to revoke Elevidys’ approval or demand a full market withdrawal, as such actions are typically reserved for fully approved drugs that have won accelerated pathway designation. Analysts warn that conducting another clinical trial in ambulatory DMD patients could set a precedent for the gene therapy space broadly.
Sarepta’s stock was down nearly 21% in Thursday afternoon trading following the news, which has sparked renewed concerns over the company’s safety and efficacy of Elevidys.
Source: https://www.fiercepharma.com/pharma/fda-weighs-new-study-requirements-sarepta-confirm-safety-gene-therapy-elevidys-report