Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Guidance for Industry; Availability

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The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance entitled "Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations." The guidance document provides FDA's current thinking on certain criteria that help determine sameness of human gene therapy products for the purpose of orphan-drug designation and orphan-drug exclusivity. The guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan-drug designation and orphan-drug exclusivity, in the development of gene therapies for rare diseases. The guidance announced in this notice finalizes the draft guidance of the same title dated January 2020.