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GUIDANCE DOCUMENT

Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations Draft Guidance for Industry January 2020

Draft

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

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Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the FDA considers your comment on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.

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

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

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


Docket Number:
FDA-2019-D-5392
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

This guidance provides FDA’s current thinking on determining sameness of human gene therapy products  under FDA’s 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.