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The draft guidance document offers developers of these treatments information regarding product development, preclinical testing, and clinical trial design.
On Jan. 5, 2021, FDA published draft guidance providing sponsors of human gene therapy (GT) products for neurodegenerative diseases information on product development, preclinical testing, and clinical trial design. Marketing approval pathways are also discussed in the document. Neurodegenerative diseases are disorders where the central nervous system or peripheral nervous system degenerates.
Specifically, the guidance addresses chemistry, manufacturing, and controls (CMC) considerations and states aspects of GT products for neurodegenerative diseases may have particular CMC considerations. The guidance emphasizes product design should be based on current scientific knowledge and GT products should be designed to reduce inflammatory immune responses. Considerations for drug product purity, identity, and strength are also emphasized.
The guidance also discusses aspects of preclinical studies, clinical trials, expedited programs, and communications with FDA.