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Human Gene Therapy for Hemophilia Draft Guidance for Industry July 2018


Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Human Gene Therapy for Hemophilia

Docket Number:
FDA 2018-D-2238
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research

This guidance is intended to assist stakeholders developing human gene therapy (GT)1 products for the treatment of hemophilia. This guidance provides recommendations on the clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. This guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia. Additional clinical and preclinical recommendations are available through several other guidances. 2,3 This guidance does not provide recommendations for products for the treatment of hemophilia C (factor XI deficiency) or for the treatment of any bleeding disorders other than hemophilia A and B, because of the unique nature of those other bleeding disorders.

Submit Comments

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:

Division of Dockets Management (HFA- 305)
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 2018-D-2238.


Contact Point
Office of Communication, Outreach and Development (OCOD)
Center for Biologics Evaluation and Research
Food and Drug Administration
10903 New Hampshire Ave WO71-3128
Silver Spring, MD 20993-0002
(800) 835-4709
(240) 402-8010