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Date: January 24-25, 2022  

Background and Meeting Objectives 

Niemann-Pick Type C (NPC) is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. NPC is caused by mutations in either the NPC1 or NPC2 genes, resulting in impaired intracellular transport of cholesterol and other lipids. Individuals with NPC have significant unmet treatment needs. Currently, there are no approved therapies in the United States for treatment of NPC.   

On January 24-25, 2022, the FDA and Duke-Margolis will host a virtual public workshop on endpoint considerations to facilitate drug development for NPC. During this workshop, participants will discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC. The workshop is open to the public and will include representation from the patient community, health care providers, industry, and other federal partners. 

For more information and to register, please visit Endpoint Considerations to Facilitate Drug Development for Nieman-Pick Type C on the Duke Margolis Center for Health Policy website.

You may submit comments for this workshop to Docket FDA-2021-N-1297. The Docket will be open until April 25, 2022. Comments in the Docket will be reviewed after the Docket closes.