Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC)


Public Workshop

Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC)


Workshop Recording Day 1

Workshop Recording Day 2

Meeting Date and Time

January 24, 2021: 12:00pm - 3:30pm ET

January 25, 2021:  12:00pm - 3:30pm ET


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.

In order to advance NPC drug development, it is important that stakeholders work together and identify strategies to support ongoing and future NPC clinical trials. In this workshop, participants will discuss clinical endpoints relevant to NPC clinical trials and innovative strategies to support therapeutic development for patients with NPC.

December 14, 2021 Update

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.



Funding for this workshop was made possible in part by a cooperative agreement from the U.S. Food and Drug Administration. The views expressed in written workshop materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.