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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 hosted a virtual public workshop on endpoint considerations to facilitate drug development for NPC. During this workshop, participants discussed clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC. The workshop was open to the public and included representation from the patient community, health care providers, industry, and other federal partners. 

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

Comments pertaining to this workshop are available at  Docket FDA-2021-N-1297. The Docket closed on April 25, 2022.