Vinay Prasad's Appointment To FDA's CBER Triggers Questions Over Future Of Cell And Gene Therapy Regulation

FDA Commissioner Marty Makary on Tuesday named Vinay Prasad the next director of the U.S. Food and Drug Administration’s (FDA) Center of Biologics Evaluation and Research (CBER).

Dr. Prasad has openly criticized the biopharma industry and the FDA for using accelerated approvals broadly based on surrogate endpoints.

“We are surprised by the announcement because it seems to be in stark contrast to themes and initiatives that have been laid out by Dr. Makary, and it comes on the heels of large personnel departures across the FDA, raising questions on the ability to meet review timelines,” writes William Blair of Blue Matrix.

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Analyst Matt Phipps adds that there are many questions about how Prasad will shape regulatory decisions as the new head of CBER. Investors also seem confused about what CBER regulates compared to the Center of Drug Evaluation and Research (CDER).

Generally, drugs like antibodies, bispecifics and antibody-drug conjugates (ADCs) are reviewed by CDER, even though they use biologics license applications (BLAs).

CBER, on the other hand, oversees gene and cell therapies, vaccines and some other biological products. CDER typically handles therapeutic antibodies, proteins, cytokines and enzymes.

Blair wrote on Tuesday that for cell and gene therapies stocks that have reacted negatively, there are outstanding questions and increased uncertainty now as the analyst waits to see whether Makary or Prasad will have more impact on the guidelines and regulatory development requirements for these novel therapies, particularly in rare diseases.

Since being confirmed as commissioner, Makary has advocated for more accelerated approval opportunities for ultrarare diseases or therapies with overwhelming efficacy.

The vaccine space continues to face negative sentiment from HHS Secretary Robert F. Kennedy Jr., who requested placebo-controlled studies for new vaccine approvals.

Prasad’s appointment at CBER will likely continue to pressure this sector.

“Under Secretary Kennedy’s leadership, all new vaccines will undergo safety testing in placebo-controlled trials prior to licensure — a radical departure from past practices,” HHS told CNN in a statement Wednesday.

Blair writes that from the coverage list, Moderna Inc MRNA has seen a sharp sell-off and remains in a difficult position with a politically out-of-favor mRNA vaccine technology and a potentially more restrictive CBER head and review process for new vaccine products.

In contrast, Dynavax Technologies Corporation DVAX remains largely insulated given that Heplisav is approved and the analyst sees less scrutiny on adult vaccines that are not being pushed as mandatory.

Taysha Gene Therapies TSHA fell 18%, Solid Biosciences Inc SLDB closed 20.3% lower, Capricor Therapeutics Inc CAPR stock closed 29% lower, Crispr Therapeutics AG CRSP stock closed 12% lower, Sarepta Therapeutics Inc SRPT stock closed 27% lower, Moderna fell 12%, Pfizer Inc PFE fell 4.2%, and Novavax Inc NVAX closed 3.2% on Tuesday. Virtus LifeSci Biotech Clinical Trials ETF BBC closed 9.74% lower at $17.15 and SPDR Series Trust SPDR S&P Biotech ETF XBI fell 6.63% to $77.17 on Tuesday.

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