The Niemann-Pick Type C (NPC) Disease Biomarker and Clinical Endpoint Initiative is a new program to that will focus on the advancement of three core themes:

i. Refine FDA and care expert recommendations to improve NPC-CSS

ii. Identify and advance candidate biomarker(s)

iii. Identify potential intermediate endpoints

The ultimate goal of the initiative will be to have candidate biomarkers and clinical endpoints for current NPC therapies and future clinical trials.

In recent years, the NPC community has been discouraged and frustrated by hurdles they have experienced regarding clinical trials and regulatory reviews of candidate therapies. On May 21, 2022, the Ara Parseghian Medical Research Fund (APRMF) organized the Niemann-Pick Type C Biomarker/Endpoint Workshop to begin a collaborative effort to address these challenges. The workshop was to build upon earlier efforts including a patient-focused drug development (PFDD) meeting in 2019, multiple listening sessions with the FDA in 2021, and a workshop hosted by the Duke-Margolis Center for Health Policy in January 2022.

The goal of the workshop was to bring together scientific, clinical, and regulatory experts to identify potential paths forward that could lead to new, effective therapies for NPC.

Please click on the link below to get access to the published report from the workshop:

The APMRF and our partners are currently supporting multiple research programs to identify candidate biomarkers and working with NPC clinicians, industry and regulators to refine and improve the NPC Clinical Severity Scale (NPC-CSS). Next steps for each core theme are outlined in more details in the Workshop report linked above.

As progress is made updates will be published on this website.