Guidance on Developing Cellular and Gene Therapy Products

Key changes

Provides specific answers to regulatory questions regarding CGT product development, including expectations for clinical monitoring and data collection. Clarifies issues related to potency, toxicity testing, and clinical safety endpoints for cell and gene therapies.

PV impact

Clinical safety and PV teams developing CGT products should review the clinical sections of this FAQ to ensure safety monitoring plans align with FDA expectations. The guidance clarifies requirements for clinical data collection and signal management in the context of advanced therapies, which often require long-term follow-up and intensive safety monitoring.

Exact policy details

Regulatory Framework

Guidance intended to help facilitate the development of safe, effective, and high-quality CGT products.

Applies to: Applicable to developers of cellular and gene therapy products.

The guidance is intended to provide industry with answers to frequently asked questions (FAQs) and commonly faced issues that arise during the development of CGT products. · p. 1 · High · Source

Non-binding recommendations

FDA’s guidance documents, including this guidance, do not establish legally enforceable responsibilities.

Applies to: General applicability to FDA guidance for Industry.

In general, FDA’s guidance documents, including this guidance, do not establish legally enforceable responsibilities. · p. 1 · High · Source

Submission requirements for IND

Commercial INDs must be submitted in eCTD format.

Applies to: For owners of commercial INDs.

A commercial IND is generally one for which the sponsor intends to commercialize the product by eventually submitting a marketing application. In this case, the sponsor should select 'Commercial IND' on FDA Form 1571. · p. 6 · High · Source

Feedback timeline

FDA has established a timeline for feedback on new or revised information submitted to an active IND.

Applies to: For sponsors of active INDs.

What is FDA's timeline for feedback on new or revised information submitted to an active IND? · p. 7 · High · Source

Submission format for non-commercial INDs

Noncommercial IND sponsors should submit their applications in common technical document (CTD) format.

Applies to: For sponsors of non-commercial INDs.

FDA recommends that noncommercial IND sponsors submit their applications in common technical document (CTD) format. · p. 7 · High · Source

Extracted documents

• FAQ— Developing Potential Cellular and Gene Therapy Productshtmlhtml · high · 1 page
• Download the Final Guidance Documentpdfpdf_text · high · 45 pages