This guidance provides recommendations to sponsors developing human gene therapy (GT) products for neurodegenerative diseases affecting adult and pediatric patients.
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 This guidance finalizes the draft guidance of the same title dated January 2021.
In general, FDA’s guidance documents do not establish legally enforceable responsibilities.Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of tthe word should in Agency guidances means that something is suggested or recommended, but not required.
Source: FDA
Docket Number:FDA-2020-D-2101
Issued by:Center for Biologics Evaluation and Research
Download Link:https://www.fda.gov/media/144886/download