Duke-Margolis NPC Endpoint Considerations Workshop Summary Now Available

Please find the workshop summary  from the Duke-Margolis NPC Endpoint Considerations Workshop is now available on the Duke-Margolis website and attached to this email. They will also be holding a brief webinar along with the FDA on Thursday, August 4, from 3:00-4:00 pm ET to provide an overview of the key themes and future directions shared during the January 2022 workshop and presented in this newly released workshop … Read More

Letter from the NPC community to Dr. McClellan, director of the Duke-Margolis Center 

On Monday, December 13th, members of the NPC community sent a letter to Dr. McClellan, who is the director of the Duke-Margolis Center that is  hosting a workshop on NPC endpoints on January 24 & 25.  The letter and response is below. It is imperative to the NPC community that the NPC- Clinical Severity Scale is a key topic of … Read More

Parseghian Fund Fall Fundraiser and Reception

Parseghian Fund Fall Reception – 2021On Friday, October 1, the Parseghian Fund’s hosted their annual Fall fundraiser and reception. It was so great to be able to see and spend time with everyone…in person! We were also grateful that attendees were able to hear from Cindy Parseghian, Santiago Schnell, and Samantha Berns. Samantha is battling NPC disease and was able … Read More

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

Pfreigers Digests – September 2021

Research at Notre Dame

Update from Mandos, LLC

Mandos is a biopharmaceutical company committed to furthering development of adrabetadex* for Niemann-Pick Type C. The company is led by a diverse and passionate management team experienced in every stage of the drug development process from research through commercialization. Please find a link to the September communication from Mandos, LLC. https://mandoshealth.com/communications.html

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

Orphazyme announces publication of results from its Phase 2/3 trial of arimoclomol in Niemann-Pick disease type C in the Journal of Inherited Metabolic Disease

Orphazyme A/S today announced that results from a Phase 2/3 trial of arimoclomol, an investigational heat-shock protein amplifier, in Niemann-Pick disease type C (NPC) have been published in the peer-reviewed Journal of Inherited Metabolic Disease (JIMD). You can find a link to the press release here and click here for the LINK to the publication.

Letter from the FDA concerning Adrabetadex Access and the Parseghian Fund’s Response

Dear Dr. Joffe and Dr. Stein, We will share your June 17 letter with the NPC community today.  However, we feel the FDA decision to allow only the early-infantile children and children currently in the EAP program to have access to Adrabetadex is not sufficient.  All children and adults should have access to this program in the spirit of transparency … Read More

Orphazyme update – FDA reviews and does not approve the NDA for Arimoclomol.

Below, please find the official statement to the Niemann-Pick Type C community about the announcement that the U.S. Food and Drug Administration (FDA), following its review of the new drug application for arimoclomol for the treatment of NPC disease, will not be approved in its current form. We are deeply saddened by this announcement and will work with Orphazyme and … Read More