Shares in more than a dozen gene and cell therapy developers tumbled by double digits Wednesday after a well-known critic of the speedy approval paths their treatments often rely on was named to lead the FDA office that regulates them.
Dr. Vinay Prasad, an oncologist and contrarian voice in clinical research, was hired by FDA Commissioner Martin Makary to run the Center for Biologics Evaluation and Research. He will succeed Peter Marks, who resigned from the FDA in March after nine years in the role.
Marks’ departure was acutely felt by the makers of gene and cell therapies, many of which are currently struggling to attract investment. He oversaw clearance of the first gene therapy, the first cellular treatment for cancer and the first CRISPR gene editing medicine, among others. He also advocated strongly for the FDA to be more flexible in reviewing those treatments, earning both praise and criticism.
“Peter Marks’ vision, scientific rigor, and outstanding clinical judgment have been key reasons that cell and gene therapies have progressed as they have,” Katherine High, a prominent gene therapy researcher, told BioPharma Dive in March. “It would be difficult to imagine a successor who could fill his shoes.”
Biotech analysts were optimistic a successor would similarly endorse the “accelerated” approval paths Marks championed, particularly with genetic medicines for rare diseases. Scott Steele, who was temporarily made acting director after Marks left, seemed to fit a similar mold.
Prasad’s appointment has upended those expectations. On the social media platform X, his YouTube channel and blog posts, Prasad has regularly criticized Marks’ decisionmaking as “pro-pharma.” He opposed Marks’ handling of the review and approvals of COVID-19 vaccines and castigated Marks’ controversial decision to overrule other FDA reviewers in clearing Sarepta Therapeutics’ Duchenne muscular dystrophy treatment Elevidys.
"Peter Marks was one of the most dangerous, pro-pharma regulators of the 21st century,” Prasad wrote in March.
Prasad previously suggested he’d “hold development” of Verve Therapeutics’ CRISPR-based treatment for high cholesterol. He has also been skeptical of the push by cell therapy developers to test their products in inflammatory disease, noting in a 2024 op-ed how it is “likely that harms may swell in decades of follow-up” in people with conditions like lupus.
Following news of Prasad’s appointment, shares of most major public gene and cell therapy developers sold off, erasing billions of dollars in market value. Some, like Sarepta and UniQure, fell by nearly 30%, while share prices of others including Verve, Rocket Pharmaceuticals and Beam Therapeutics declined by almost 20%.
Multiple Wall Street analysts saw the change in leadership adding uncertainty to an already struggling field, though it’s unclear to what degree. Jefferies analyst Michael Yee wrote that Prasad’s views “seems to fly in the opposite barometer” of Marks’. William Blair analyst Matt Phipps called the announcement a “surprise” given it’s in “stark contrast to the themes and initiatives that have been laid out” by Makary, such as his apparent support of faster approvals for certain rare disease treatments.
“There are clearly outstanding questions and increased uncertainty now, as we wait to see whether Dr. Makary or Dr. Prasad will have more impact on the guidelines and regulatory development requirements for these novel therapies, particularly in rare diseases,” Phipps wrote.
To Andrew Tsai, another Jefferies analyst, the FDA can "still be supportive” of accelerated approvals with Prasad running CBER, companies just may need to “produce more supporting clinical evidence” and take “less ‘shortcuts.’”
Brian Skorney, of Baird, added that it may be much harder going forward for companies to “avoid accountability” with confirmatory studies. Pulling products from the market when they fail could become more common, too, he wrote.
“Cell and gene therapy now face a new challenge regarding the bar for full approval and the use of bioplausibility in granting accelerated approval,” Skorney added.
Jacob Bell contributed reporting.
