- Docket Number:
- FDA 2018-D-2238
- Issued by:
Guidance Issuing OfficeCenter for Biologics Evaluation and Research
This guidance provides recommendations to sponsors developing human gene therapy (GT) products for the treatment of hemophilia including 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 in other guidances (Refs. 1 and 2). 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 bleeding disorders. This guidance finalizes the draft guidance of the same title dated July 2018.
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:
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.