On August 3, 2021, the Ara Parseghian Medical Research Fund (APMRF), International Niemann-Pick Disease Alliance (INPDA), and National Niemann-Pick Disease Foundation (NNPDF) convened a listening session with representatives from the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Review (CDER) Office of New Drugs (OND). This session, which was held at the request of the FDA, gave members of the Niemann-Pick Type C (NPC) community an opportunity to share thoughts on two key topics: the relative benefits and risks of NPC treatment options and the value of the NPC Clinical Severity Scale (CSS) in measuring disease progression. The session featured remarks from expert NPC clinicians, patients and family members, and NPC advocacy organization representatives (see agenda in Appendix A). Although titled a “listening session,” FDA staff were active participants in the session, responding to remarks from NPC community members and sharing their thoughts on NPC therapy development.
The entire report can be found on the link below: