On January 11, 2026, the FDA announced information about the agency’s flexible approach to overseeing chemistry, manufacturing and control (CMC) requirements for cell and gene therapies (CGT). The agency’s more flexible approach has been, and is expected to continue to be, helpful in expediting product development and will help guide the FDA’s evaluation of development strategies in preparation for a Biologics License Application (BLA) submission.
1. Clinical Development Flexibilities
Before an investigational product is manufactured for phase 2 or 3 trials, the manufacturer will not be expected to comply with 21 CFR part 211. See 21 CFR 210.2(c).
The FDA reviews process and method validation consistent with a product lifecycle approach, understanding that process and method validations approaches are refined over time.
As final specifications for the drug substance and drug product are not expected until the end of the investigational process (21 CFR 312.23(a)(7)), INDs may provide for permissive product quality release acceptance criteria in investigational studies consistent with the quality requirements for products used in investigational studies.
As sponsors move from phase 1 to studies designed to establish efficacy for licensure, CBER will allow minor manufacturing changes supported by data showing the comparability of the pre-change and post-change product without expecting overly stringent and onerous comparability data.
2. Commercial Specifications Flexibilities
The FDA’s approach is based on the need to ensure product quality and compliance while also recognizing that the small patient populations targeted by CGT therapies do not necessarily allow for a large number of manufacturing lots to support the product release specifications.
CBER will consider, where appropriate, flexibility in establishing product release specifications in the review of CGT BLAs, consistent with the nature of the product and process and when appropriately justified.
CBER will consider submissions seeking to re-evaluate and revise product release acceptance criteria based on post-approval manufacturing experience, when manufacturers demonstrate consistent product quality.
3. Process Validation Flexibilities for CGT
There may be situations when specific Process Performance Qualification (PPQ) lots can be designed by batch for release and distribution before protocol execution steps are completed (i.e. concurrent release).
There is no requirement to supply three (3) PPQ lots for process validation.
FDA’s review may consider whether PPQ protocols justify the appropriate number of lots based on overall process understanding.
FDA is cognizant of the rapid pace of scientific advancements related to the development and manufacturing of CGT products, and we encourage sponsors with questions about CMC requirements to consult with review divisions throughout the product development process.
Source from FDA
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