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US: New OTP head discusses policy priorities for cell and gene therapies

2023/09/21  RAPS

The new director of the US Food and Drug Administration’s Office of Therapeutic Products (OTP), within the Center for Biologics Evaluation and Research (CBER), supports the use of platform designations for approving new cell and gene therapy treatments and wants to shift back to communicating directly with sponsors.

Nicole Verdun, MD, in the opening address at the 18 September meeting of the American Society of Gene and Cell Therapy (ASGCT), discussed her policy priorities going forward. OTP, formerly called the Office of Tissues and Advanced Therapies (OTAT) was renamed in September 2022 and reorganized to a “Super Office” to meet its growing workload and new commitments under the Prescription Drug User Fee Act (PDUFA VII) agreement.

The session’s moderator, Kristin Van Goor, PhD, of Takeda, said she sees “amazing opportunities” in the gene editing space where the technology can target specific mutations. “If you go mutation by mutation you are going to be doing it forever,” she said. “With a platform you would be able to bundle these things together, especially in the rare disease space, this could be transformational.”

Verdun agreed, calling it an “exciting area” that offers “a lot of opportunities” for cell and gene therapies. She said a platform approach could be used in the gene therapy area, such as using the same adeno-associated virus (AAV) backbone “to move more than one product forward at any given time.”

The omnibus spending bill approved by Congress in December 2022 directs FDA to establish a platform designation for drugs or biological products that have technologies that can be incorporated in or used by more than one drug or biological product, and which can make the development and manufacturing process and the review more efficient.

Currently, the movement toward a platform designation “is more of an agency wide initiative, but there are things that are specific to cell and gene therapies, and this is something that could be very exciting and could help move the field forward,” Verdun said.

Moving to other topics, Van Goor asked Verdun to describe her vision for how the division plans to communicate with sponsors going forward.

Verdun said she supports more informal and verbal communications with sponsors. “I think during the pandemic the communication piece changed a lot. We are tasked with the challenge of shifting things back to more communications with sponsors and less of the written response only.”

She added that OTP is “committed to decreasing the number of written responses” and that in the future the agency envisions “a shift from written only responses to more interactive responses.”

Another question targeted how sponsors could improve their cell and gene therapy submissions. Verdun said that one area that can be improved is for sponsors to heed the agency’s advice, especially in the preclinical stage.

“Some sponsors just look at that advice and ignore it. There might be a good reason why they can’t take that advice. If you’re coming in with an application that completely ignores the advice without any communication in between, that does not foster the type of thing that we need to move your product forward,” she said. “That is something that we do see.”

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