NPC Listening Session with the FDA on August 3rd – Final Report

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 … Read More

Letter from the NPC community following the FDA Listening Session on August 3, 2021

August 13, 2021 Kathleen Donohue, MD Food and Drug Administration Center for Drug Evaluation and Research Office of New Drugs Division of Rare Diseases and Medical Genetics 10903 New Hampshire Avenue Silver Spring, MD 20993 RE: August 3, 2021 FDA-NPC Community Listening Session Dear Dr. Donohue: On behalf of the Ara Parseghian Medical Research Fund, the National Niemann-Pick Disease Foundation, … Read More

Notes from NPC Accelerator Meeting – 5-26-21

AttendeesSean KassenJoslyn CrowePhil MarellaChris AndrewsCristin DavidsonDave Zook Topics- Mandos acquisition of Adrabetadex – Discussed outreach with Mandos and connecting them to the community. We must wait until the bankruptcy court approves the deal Orphazyme update – PDUFA date for Arimoclomol is June 17. Waiting to learn more about the pending review/approval. NNPDF helped to get letters to send to the … Read More

Notes from NPC Therapy Accelerator Meeting – 3/16/2021

Sean Kassen, APMRFJustin Hopkin, NNPDFPhil Marella, Danas Angels Research Trust, NPC ParentCristin Davidson, NPC ScientistDave Zook, Faegre DrinkerTim Franson, Faegre Drinker The main topic of the conversation was the Parseghian Fund had a 1 hr meeting with Mallinckrodt Pharmaceuticals, which included Elissa Cote, Josh Schafer, and Vadim Rechanskiy and below are points that were shared with the team: Mallinckrodt has … Read More

Message from the NTA to Mallinckrodt in regards to Adrabetadex


Dear Steve and Elissa, I am writing on behalf of the Niemann-Pick Type C (NPC) Therapy Accelerator in regard to the adrabetadex program.  Specifically, we would like to set up a time to speak with Mallinckrodt (MNK) about the future of adrabetadex, and discuss possibilities for allowing the NPC community to gain access to the investigational product and the related … Read More

NTA Publishes Survey on Preferences for Treatments for NPC Disease

On behalf of the Niemann-Pick Type C Therapy Accelerator (NTA) team, we would like to share with you a recent patient and caregiver survey in order to better understand the experiences of people with Niemann-Pick Type C (NPC) and their preferences for future treatments.  This survey included questions about NPC symptoms, treatments, the impact of the disease on daily life, … Read More

Letter from the NTA to the FDA announcing the formation of the Accelerator

Dear Dr. Woodcock and Dr. Donohue: As leaders across the Neimann-Pick Type C (NPC) Community, we are writing to inform you of a significant collaboration among our organizations aligning efforts to help bring treatments to patients and families.  The NPC Therapy Acceleration Initiative will ensure a strategic and coordinated approach to our shared objectives.  The externally-led Patient-Focused Drug Development Meeting … Read More