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Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment Guidance for Industry Draft Guidance for Industy February 2020

Download the Draft Guidance Document Read the Federal Register Notice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Not for implementation.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).

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5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-5404.

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