Webinar on Endpoint Considerations to Facilitate Drug Development for Niemann-Pick Type C (NPC): Key Themes and Future Directions from the January 2022 Public Workshop

Niemann-Pick Type C (NPC) is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. NPC is caused by variants in either the NPC1 or NPC2 gene, 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 January 2022, the Robert J. Margolis, MD, Center for Health Policy at Duke University and the U.S. Food and Drug Administration (FDA) convened a group of experts to discuss clinical endpoints relevant to clinical trials and innovative measurement strategies with the overall goal of supporting the development of safe and effective treatments for those living with NPC. This follow-up webinar will provide an overview of the key themes and future directions shared during the January 2022 workshop and presented in the recently released workshop summary report.