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GUIDANCE DOCUMENT

Human Gene Therapy for Neurodegenerative Diseases Guidance for Industry October 2022

Download the Final Guidance Document Read the Federal Register Notice
Final
Docket Number:
FDA-2020-D-2101
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients.  Neurodegenerative diseases are a heterogeneous group of disorders characterized by progressive degeneration of the structure and function of the central nervous system or peripheral nervous system.  These diseases vary in etiology, prevalence, diagnosis, and management, and include genetic as well as age-related diseases.   This guidance focuses on considerations for product development, preclinical testing, and clinical trial design.  This guidance finalizes the draft guidance of the same title dated January 2021.

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All written comments should be identified with this document's docket number: FDA-2020-D-2101.

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