GUIDANCE DOCUMENT

Draft

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

This guidance document provides recommendations for developing assays  to assess potency  as a part of a potency assurance strategy for active immunotherapy products (ACTIMPs) .  Active immunotherapies aim to treat a pre-existing disease or condition by inducing, stimulating, or modulating immune effector cells through introduction of an antigen (or antigens) associated with that disease or condition to the immune system.  This guidance does not apply to preventive vaccines for infectious disease indications, bacteriophage products for infectious diseases, live biotherapeutic products, fecal microbiota for transplantation (FMT) products or allergenic products.  Considerations related to potential safety concerns associated with ACTIMP product design are outside the scope of this guidance. Sponsors are therefore encouraged to consult with the appropriate product office regarding such matters.

When finalized, this guidance will describe the FDA’s current thinking regarding design, validation, and evaluation of potency tests for ACTIMPs.  This guidance is intended to supplement the guidance for industry, when finalized, on Potency Assurance for Cellular and Gene Therapy Products (Ref. 1) with additional advice and considerations specifically for ACTIMPs, including peptide- and-protein based ACTIMPs that are not cellular or gene therapy products (Ref. 2).