U.S. flag An official website of the United States government
  1. Home
  2. Regulatory Information
  3. Search for FDA Guidance Documents
  4. Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment Guidance for Industry
  1. Search for FDA Guidance Documents

GUIDANCE DOCUMENT

Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment Guidance for Industry Draft Guidance for Industy February 2020

Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

Submit Comments by

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can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the FDA considers your comment on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.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-5404


Docket Number:
FDA-2019-D-5404
Issued by:
Guidance Issuing Office
Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research
Office of Medical Products and Tobacco, Center for Drug Evaluation and Research

The purpose of this guidance is to provide recommendations to sponsors regarding eligibility criteria, trial design considerations, and efficacy endpoints to enhance clinical trial data quality and foster greater efficiency in development programs for drugs to treat mucopolysaccharidosis type III (MPS III; also called Sanfilippo syndrome).