The departure of the US Food and Drug Administration’s (FDA) cell and gene therapy director and deputy director fuels ongoing turbulence in the treatment modality’s sector and could lead to changes in safety monitoring for drugs, according to an investment analyst.
Dr Nicole Verdun, director of the FDA’s Office of Therapeutic Products (OTP), and her deputy Dr Rachael Anatol, were placed on administrative leave on 18 June. The OTP is part of the Center for Biologics Evaluation and Research (CBER) and responsible for reviewing applications cell and gene therapy products.
“Overall, while we view the departure of Drs. Verdun and Anatol as another sentiment hit and source of volatility for the cell and gene therapy spaces,” said William Blair analyst Sami Corwin in a research note.
Corwin added that Dr. Verdun, who became OTP director in July 2023, is highly respected by industry and academic experts.
The Alliance for Regenerative Medicine, an advocacy organisation for advanced therapies, said it was “deeply disappointed” by the news, commenting that both Dr Verdun and Dr Anatol have “modernised the FDA’s regulatory approach to cell and gene therapy, earning the trust and respect of the [therapy’s] community.”
There was no reason given for their departure, although shakeup comes at a time of increased governmental scrutiny on the manufacturing and safety of advanced medicinal products.
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By GlobalDataOn a recent podcast as noted by William Blair’s Corwin, FDA commissioner Dr Marty Makary and CBER director Dr Vinay Prasad discussed that some CAR-T products, a type of immunotherapy, are being made in China for American patients, both viewing this as a “national security threat.” Corwin said that all CAR-T companies under its remit at least make products in the US or UK.
Safety reviews are also on the FDA’s agenda, with Dr Prasad stating the agency will look to use big data for enhanced safety signal analysis. Cell and gene therapy safety sadly hit the news for another reason this week, when Sarepta’s Elevidys (delandistrogene moxeparvovec) was linked to a second patient death.
However, generally, Dr Makary and Dr Prasad, along with US health secretary Robert F Kennedy (RFK) Jr have been vocal in their support for cell and gene therapies. Unlike vaccines, the agency leaders see the modality as a path to prevent and cure disease – aligning with the current administration’s wider national health strategy. Earlier this month, RFK Jr said the US department of Health and Human Services (HHS) would look at new ways of fast-tracking approvals for cell and gene therapies for rare diseases.
However, the departure of both the OTP director and deputy director signals that the FDA will likely implement new regulatory requirements going forward.
Corwin commented: “We do not ultimately think these departures from the agency will significantly impact registrational trial designs and packages that have already been aligned on, but we believe the FDA could require more stringent post-marketing safety monitoring and reporting and/or increase the requirements for patient disclosures and consent regarding data handling and safety.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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