Interpreting sameness of gene therapy products under the orphan drug regulations

Interpreting sameness of gene therapy products under the orphan drug regulations

Collection:
Health Policy and Services Research
Series Title(s):
Guidance for industry
Contributor(s):
United States. Department of Health and Human Services, issuing body.
United States. Food and Drug Administration, issuing body.
United States. Food and Drug Administration. Office of Clinical Policy and Programs. Office of Orphan Product Development, issuing body.
Center for Biologics Evaluation and Research (U.S.), issuing body.
Publication:
Silver Spring, MD : Center for Biologics Evaluation and Research, September 2021
Language(s):
English
Format:
Text
Subject(s):
Genetic Therapy
Orphan Drug Production -- legislation & jurisprudence
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration
Genre(s):
Technical Report
Abstract:
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. This guidance finalizes the draft guidance of the same title dated January 2020.
Copyright:
The National Library of Medicine believes this item to be in the public domain. (More information)
Extent:
1 online resource (1 PDF file (ii, 4 pages)).
NLM Unique ID:
9918351277306676 (See catalog record)
Permanent Link:
http://resource.nlm.nih.gov/9918351277306676