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)

January 24, 2022 12:00PM - January 25, 2022 3:30PM


Contact Information

Luke Durocher


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.



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.