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. 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.
Following the session, there are still clear disagreements between the FDA and NPC clinicians, sponsors, and patients. In particular, the community remains concerned about the recent lack of approval of new therapies and limits placed on Expanded Access Programs (EAP)—despite trial evidence which they view as showing benefits exceeding risks—and the FDA’s seeming concerns about the NPC CSS.
It is essential that this session spur additional interactions to resolve these disagreements with the ultimate goal of advancing NPC therapy development and allow for patient access in the near future.
The complete report can be found on the link below: