Interpreting Sameness Gene Therapy Products Orphan Drug Regulations – FDA.gov

Docket Number: FDA-2019-D-5392 Issued by:

Guidance Issuing Office

Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research

This guidance provides FDAs current thinking on determining sameness of human gene therapy products under FDAs orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity. This 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. This guidance focuses specifically on factors that FDA generally intends to consider when determining sameness for gene therapy products and does not address sameness determinations for other types of products.

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets ManagementFood and Drug Administration5630 Fishers Lane, Rm 1061Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-5392 .

01/28/2020

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Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov