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GUIDANCE DOCUMENT

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases Guidance for Industry December 2017

Download the Final Guidance Document Read the Federal Register Notice
Final
Docket Number:
FDA-2017-D-6380.
Issued by:
Guidance Issuing Office
Office of the Commissioner, Office of Clinical Policy and Programs, Office of Orphan Products Development

This guidance is intended for sponsors of drugs and biological products (hereafter drugs1) who are considering submitting requests for orphan-drug designation for their drugs under section 526 of the Federal Food, Drug, and Cosmetic Act (FD&C Act).  The Food and Drug Administration (FDA) does not expect to grant any additional orphan-drug designation to drugs for pediatric subpopulations of common diseases (i.e., diseases or conditions with an overall prevalence of 200,000 or greater). Pediatric-subpopulation designations that have already been granted will not be affected by this change.

Additional copies are available from:
Office of Orphan Products Development
Office of the Commissioner
Food and Drug Administration
10903 New Hampshire Ave., Bldg. 32, Rm. 5295
Silver Spring, MD 20993
Tel: 301-796-8660; E-mail: [email protected]
https://www.fda.gov/orphan

Submit Comments

Submit Comments Online

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 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-2017-D-6380..

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