U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  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Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment Guidance for Industry Draft Guidance for Industy February 2020

Download the Draft Guidance Document Read the Federal Register Notice
Draft Level 1 Guidance

Not for implementation. Contains non-binding recommendations.

Docket Number:
FDA-2019-D-5404
Issued by:
Guidance Issuing Office
Center for Biologics Evaluation and Research
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).

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-2019-D-5404.

Back to Top