Mucopolysaccharidosis Type III (Sanfilippo Syndrome): Developing Drugs for Treatment Guidance for Industry

On 4 February 2020 the FDA announced the availability of the draft guidance for industry. Comments may be submitted by 3 April 2020.

The purpose of this draft guidance is to foster greater efficiency in drug development in this rare disease with the goal of enhancing clinical trial data quality and supporting the development of treatments for mucopolysaccharidosis type III. Specifically, the draft guidance provides the Agency's current recommendations regarding eligibility criteria, trial design considerations, and efficacy endpoints for use in clinical development programs of investigational drugs to treat mucopolysaccharidosis type III.

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