FDA increases flexibility on cell and gene therapy manufacturing requirements.

The FDA said it will share its flexible approach to chemistry, manufacturing and control (CMC) requirements for cell and gene therapies to guide Biologics License Application evaluations and expedite development. The agency's Center for Biologics Evaluation and Research has approved close to 50 such therapies over the last decade and will communicate flexibilities that were previously applied case-by-case.

The U.S. Food and Drug Administration announced it is sharing information about a more flexible approach to chemistry, manufacturing and control (CMC) requirements for cell and gene therapies. The agency said the approach has been applied case-by-case and is expected to continue to expedite product development. The Center for Biologics Evaluation and Research (CBER) has approved close to 50 cell and gene therapies over the last decade. The FDA emphasized that flexibilities are intended to accommodate unique therapy characteristics while maintaining standards for safety, purity and potency. Key details: - The FDA will share its flexible approach to CMC requirements for cell and gene therapies. - CBER has approved close to 50 cell and gene therapies in the past decade. - The agency expects these flexibilities to help expedite product development and to guide BLA evaluations. - Flexibilities were previously applied case-by-case and the FDA plans to communicate them more broadly so sponsors understand scientifically acceptable approaches. - The FDA stated these measures are intended not to compromise assurance of safety, purity or potency. Summary: The agency intends these steps to clarify regulatory options for developers and to guide evaluation of development strategies ahead of Biologics License Application submissions. The FDA will communicate the CMC flexibilities more broadly and has engaged experts through forums such as a Cell and Gene Therapy Roundtable.