FDA Issues Detailed Guidance on Development of Gene Therapy Products

Earlier in November, the FDA Center for Biologics and Research (CBER) released a new FAQ for developers of cellular and gene therapy (CGT) products. Sponsors of CGT products planning INTERACT, Type-D, Pre-IND or pre-BLA meetings with the Agency will benefit from starting their question development with a detailed review of this guidance. 

It covers the differences between these meeting types while also answering several, CMC, non-clinical, and clinical questions that commonly arise during development of CGT. The guidance also provides some additional detail on IND content, cross-referencing strategies and review which will be helpful in gap analysis and submission planning. 

Additionally, clinical trial design is covered with content related to overall design, surrogate endpoints, safety data requirements and required number of trials for approval covered at a high level with reference to more detailed guidance where appropriate.    

Having all this information in one place will help sponsors in selecting the right meeting type and developing right set of questions at every stage of development. However, each GCG program is unique and will rely on resolution of several questions that fall outside even a detailed guidance such as this one. Ensuring those unique questions are addressed adequately is something we pride ourselves on at MMS so, please enquire if you are looking for some additional support.