FDA describes agency’s initiatives to accommodate growth in cell and gene therapy fields

BOSTON – A US Food and Drug Administration (FDA) official discussed the agency’s recent efforts to spur the development of cell and gene therapies during a workshop at the Drug Information Association’s (DIA) Global Annual Meeting on 27 June.
 
Kimberly Schultz, the chief of Gene Therapy Branch 4 in the Office of Therapeutic Products (OTP) discussed these efforts and how the agency is expanding its outreach to improve the quality of applications being submitted to the agency.
 
To date, FDA has approved a total of 30 cell and gene therapies, a jump from the 23 approved in 2022.
 
To facilitate the development of these therapies, FDA will can apply its expedited pathways, such as Regenerative Medicine Advanced Therapy Designation (RMAT) designation, breakthrough therapy designation (BTD) and accelerated approval.
 
Recent efforts to spur such development efforts include a recent office reorganization, which provides “additional flexibility in our ability to respond to the growth that is happening in the field and to better able to meet the needs of patients and developers.”
 
OTP, formerly called the Office of Tissues and Advanced Therapies (OTAT) was renamed last September and reorganized to a “Super Office” to meet its growing workload and new commitments under the Prescription Drug User Fee Act (PDUFA VII) agreement. (RELATED: FDA elevates OTAT to “Super Office” within CBER, Regulatory Focus 30 September 2022)
 
“This new structure will allow us over the next couple of years to expand the number of reviewers that we have and our ability to meet the needs of each individual application.”
 
Another way to bolster the development of these therapies is through the OTP town halls, which allows the agency to expand its outreach to sponsors and address questions. These town halls occur every two months and meetings rotate among the various review disciplines, including the chemistry, manufacturing and controls (CMC) and the clinical review teams.
 
The town hall series was announced in 2021 by outgoing OTAT Director Wilson Bryan to improve communications with sponsors and to widen its messaging. (RELATED: FDA explains plans to bolster cell and gene therapy approvals through wider messaging, Regulatory Focus 19 May 2022)
 
Another initiative is the CMC development and readiness pilot, which was announced in October 2022 to expedite the development of breakthrough products that address an unmet medical need through providing product-specific CMC advice during product development. (RELATED: FDA announces CMC review pilot got drugs with expedited development, Regulatory Focus 1 November 2022)
 
“We have started to take applications” for the program, she said. Nine cell and gene therapy products will be accepted.”
 
Schultz added that the clinical development for these products is “quite rapid,” and that the division is “still seeing the CMC fall behind and this is seen in the number of information requests that we see.”
 
Schultz said that helps to have more interactions between the different regulatory agencies, especially “if FDA expects one thing and the EMA or Japan the opposite,” when asked whether FDA plans to harmonize its regulatory requirements with other health authorities.
 
She added that “we are moving in the right direction” with the recent adoption of the International Council on Harmonization’s S12 guidance.
 
The guideline, which was adopted this year, provides considerations for the design, timing and conduct of nonclinical biodistribution (BD) considerations for gene therapy products and offers various recommendations for sponsors on the animal species or model to be used, the group size and sex of study animals, route of administration and dose selection, as well as sample collection. (RELATED: ICH adopts S12 guideline for gene therapies, Regulatory Focus 20 March 2023)
 
DIA Annual Meeting