Background: All eyes on the FDA’s Office of Therapeutic Products

• The Office of Therapeutic Products, or OTP, is responsible for the review of cellular therapies, gene therapies, therapeutic vaccines, and plasma-derived and coagulation products. Formerly known as the Office of Tissues and Advanced Therapies, the office was reorganized and promoted to Super Office status in 2022 to “improve functional alignment, increase review capabilities, and enhance expertise on new cell and gene therapies.” [Read Agency IQ’s breakdown of the reorganization here.]
• Director NICOLE VERDUN led OTP’s six sub-offices, 14 divisions, and 33 branches until recently. On June 18, 2025, STAT News reported that Verdun and deputy director[KO1]  RACHAEL ANATOL, were placed on administrative leave by CBER Director VINAY PRASAD and escorted off the FDA’s White Oak campus. Endpoints News subsequently reported that OTP would be led on an acting basis by VIJAY KUMAR, a nephrologist who has been at the agency since 2020.
• OTP at a crossroads: Reports linked the abrupt departure to tensions regarding Capricor Therapeutics’ deramiocel for Duchenne muscular dystrophy. Just days before news of Verdun’s separation, the agency had withdrawn a pending Federal Register notice announcing that the deramiocel application would be considered by the agency’s advisors [See AgencyIQ analysis here.]. At that time, AgencyIQ surmised that the move signaled increased activity behind the scenes. The firm subsequently announced on July 11 that its application received a Complete Response Letter from the FDA. Also on July 11, Ultragenyx announced that the FDA had issued a CRL for its gene therapy product, UX111, for certain patients with Sanfilippo syndrome Type A due to manufacturing concerns. Coupled with leadership changes, the two actions have left the CGT industry wondering whether a change in regulatory approach could be underway.

Now, the FDA has announced a forthcoming OTP public listening meeting on CGT development and review.

• Titled “Leveraging Knowledge for Facilitating the Development and Review of Cell and Gene Therapies,” the event has not been formally announced by the agency. A new FDA webpage states that it will take place September 18, 2025, from 10:00 a.m. – 4:00 p.m. EDT. Unlike recent public meetings that have been streamed widely, this event requires registration, which will “automatically close once capacity is met.” Stakeholders who wish to speak or present must request to do so before Friday, August 8.
• The agency committed to hosting this event through the seventh authorization of the Prescription Drug User Fee Act. [See AgencyIQ’s PDUFA VII deliverable tracker here.]. The FDA agreed to “convene a public meeting to solicit the perspective of cell and gene therapy manufacturers on how individual sponsors might leverage internal prior knowledge and public knowledge, including Chemistry, Manufacturing, and Controls, non-clinical, and clinical knowledge, across therapeutic contexts in order to facilitate product development and application review,” (page 56). The event will take place just a few weeks before its September 30 due date.
• Biologics developers face challenges in leveraging existing information relative to drug developers. In a new drug application, a sponsor can incorporate proprietary information from another manufacturer into its application by referencing a Drug Master File. However, the FDA’s longstanding policy is that biologics license applications generally may not rely on drug substance, drug substance intermediate, or drug product information contained in master files. The agency has explained that this practice is “based on the differences in risk.” For biologics, references are limited to information on manufacturing, processing, packaging, or storing.
• The meeting webpage says that the event consists of three sessions that provide CMC, nonclinical, or clinical perspectives. The agency lays out four questions to be considered across these perspectives: (1) What types of data and information are sponsors willing to share that will be useful for advancing CGT product development and regulatory review? (2) What data leveraging is possible between external partners (such as contract manufacturing organizations, contract development and manufacturing organizations, and licensees) while considering various aspects of product lifecycle? (3) How can data and information be effectively integrated and leveraged across multiple disciplines to enhance the development, manufacturing, and safety assessment of CGT products? And (4) What mechanisms could be used to facilitate data and information sharing?
• Intentional data sharing in CGT development was a key talking point of Verdun at a May 2025 public meeting hosted by the Friends of Cancer Research and the Parker Institute for Cancer Immunotherapy. From a review standpoint, she emphasized that the agency is “very comfortable in a space where we allow leverage across programs, if it makes sense.” For example, prior to having an approved therapy, the office sees “backbones that are being used across six, seven different indications” that leverage common nonclinical and CMC information. [Read full AgencyIQ analysis of that meeting here.].
• According to the PDUFA Commitment Letter, the input gained at the meeting will inform guidance development, with a draft guidance to be published by Sept. 30, 2026. The FDA agreed to issue a revised draft or final guidance within 18 months of the close of the public comment period, which would correspond with mid-2028.

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