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.
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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.”
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By GlobalDataNawab 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.
“While it is unclear how much influence Dr. Prasad had on CBER’s regulatory actions in recent months, the contentious regulation of Elevidys was particularly public, suggesting the optics of it, and its reflection on FDA more broadly, may have played a role in Dr. Prasad’s departure from the agency,” William Blair analysts stated.
An HHS spokesperson told Pharmaceutical Technology: “Dr. Prasad did not want to be a distraction to the great work of the FDA in the Trump administration and has decided to return to California and spend more time with his family. We thank him for his service and the many important reforms he was able to achieve in his time at FDA.”
Prasad, an oncologist who was a fierce critic of the Covid-19 vaccine response in the US, was appointed only three months ago.
Major shakeups that occurred on Prasad’s watch included leadership changes within departments of the CBER. The director of the Office of Therapeutic Products (OTP), Nicole Verdun, was placed on administrative leave in June. Verdun is a highly respected individual in the cell and gene therapy space by academics and industry experts. Her departure was seen a knock to the future of cell and gene therapies – the Alliance for Regenerative Medicine, an advocacy organisation for advanced therapies, said it was “deeply disappointed” at the time.
Major changes to the Covid-19 vaccine recommendations were also approved under his tenure, such as limiting booster doses in certain adults.
It is unclear who will replace Prasad as head of CBER. Given FDA chief Marty Makary’s views closely aligned with those of Prasad, it is unlikely there will be significant deviation in regulatory policies. However, analysts are concerned that a lack of continuity in leadership may impact the FDA’s ability to review timelines. Health secretary Robert F Kennedy Jr has said in June the FDA would look at ways of fast-tracking approvals for drugs treating rare diseases, though no update has been issued since.
“Prasad was known for challenging FDA orthodoxy, particularly the accelerated approval pathway used in the Sarepta (Elevidys) decision. His exit raises critical questions: which evidence standards will prevail: traditional or accelerated?” commented Cynthia Pussinen, CEO of Owl Bio Advisors, a life sciences consultancy group.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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