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FDA Adopts Flexible CMC Oversight for Cell and Gene Therapies

The update details targeted allowances across development stages to speed access without compromising product quality.

Overview

  • The FDA’s January 11 update commits to applying tailored CMC flexibility across clinical development, BLA review, and process validation for cell and gene therapies.
  • Manufacturers are not expected to meet 21 CFR part 211 CGMP requirements before producing investigational products for phase 2 or 3 trials.
  • INDs may use permissive release acceptance criteria, and minor manufacturing changes can proceed during development when supported by comparability data.
  • During BLA review, the agency will consider justified flexibility in commercial release specifications and may later re-evaluate acceptance criteria based on post-approval experience.
  • The FDA outlined validation flexibilities, including possible concurrent release and waiving the traditional three PPQ-lot expectation when the proposed lot count is justified by process understanding.