Vinay Prasad, head of the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA), has stepped down, with market analysts forecasting a boost to the cell and gene therapy space.
Prasad, while a supporter of cell and gene therapies, was critical of using surrogate endpoints as evidence for accelerated approvals. A surrogate endpoint is a clinical trial endpoint used as a substitute for a direct measure of how a patient feels, functions, or survives. They can be important in streamlining reviews for therapies where clinical endpoints are slow or difficult to measure.
Prasad’s departure, confirmed to Pharmaceutical Technology by a department for Human Health and Services (HHS) spokesperson, could be a boost for the cell and gene therapy space, as per market analysis.
“Based on his prior criticisms regarding the use of accelerated approvals based on surrogate endpoints, Dr. Prasad’s departure may alleviate some of the overhang on the cell and gene therapy spaces, where accelerated approval pathways are often used,” said William Blair analysts in a 30 July research note.
The shakeup at the FDA comes in the same week as Sarepta faced intense regulatory scrutiny over its Duchenne muscular dystrophy (DMD) gene therapy Elevidys. Following the third death being reported, the FDA requested that Sarepta voluntarily pause shipments of Elevidys to all commercial patients on 18 July. Despite a brief rebuff, Sarepta eventually agreed to the agency’s request. The agency then opened an investigation linking the drug to a third death, which was swiftly resolved and placed Sarepta in the clear.
GlobalData healthcare analyst Asiyah Nawab commented: “Prasad, a vocal critic of accelerated approvals, had taken a firm stance against therapies like Sarepta’s Elevidys, which was greenlit despite limited efficacy data and lingering safety concerns, including patient deaths. His departure follows the recent failure of Capricor’s DMD gene therapy, further fuelling uncertainty in an already volatile market.”
Nawab added: “While Prasad’s exit could open the door for a more permissive FDA approach, potentially giving Capricor a path back into the market, it’s also possible that someone equally or even more conservative could take over, closing that window entirely. The future of DMD drug development now hinges on who steps into the role and how the FDA navigates this high-profile therapeutic area.”
The public playout of DMD treatment developers with the FDA is not only impacting biopharma share prices but also raising concerns about the agency’s internal operations.
