The Food and Drug Administration released a final guidance on Monday that provides recommendations to sponsors of human gene therapy products for neurodegenerative diseases affecting adult and pediatric patients. The guidance, titled “Human Gene Therapy for Neurodegenerative Diseases,” focuses on considerations for product development, preclinical testing and clinical trial design. It finalizes the draft guidance of the same title dated January 2021.
FDA is accepting comments on the final guidance at any time. Comments should include the document's docket number (FDA-2020-D-2101).
