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
Mandos, LLC to acquire Adrabetadex from Mallinckrodt
It is with much enthusiasm we announce that Mallinckrodt Pharmaceuticals reached an agreement to divest and transfer the IND application of Adrabetadex to the company, Mandos, LLC. With this news, it is their goal to continue to develop the therapy and provide access of Adrabetadex up to an additional two years to eligible patients. Here is a link to the press release. … Read More
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
Hear from Clinicians involved in the Adrabetadex Program – Webinar featuring Dr. Denny Porter and Dr. Elizabeth Berry-Kravis
You are invited to join a webinar featuring Dr. Elizabeth Berry-Kravis and Dr. Denny Porter who will share information on the Adrabetadex Expanded Access Program, the historical data collected by Dr. Porter and others in regards to the Adrabetadex program, and an overview of the NPC Natural History Data. We also feel strongly that the patient’s voice is part of the process … Read More
Adrabetadex update webinar
Please find the video recording from the January 22, 2021 webinar where Mallinckrodt Pharmaceuticals presented their reasoning behind the discontinuation of the Adrabetadex program.
Adrabetadex update from Mallinckrodt – Jan 2021
Please read the below letter that was shared from Mallinckrodt Pharmaceuticals this morning. Please note we will have a Zoom Webinar on Friday, January 22, at 12:30 – 1:30 pm to hear from Dr. Steve Romano, Executive Vice President and Chief Scientific Officer at Mallinckrodt Pharmaceuticals and Dr. Susan VanMeter, Senior Director, Clinical Research at Mallinckrodt Pharmaceuticals to discuss their plans in regards … Read More
Adrabetadex update from Mallinckrodt
Please join Dr. Steve Romano, Executive Vice President and Chief Scientific Officer at Mallinckrodt Pharmaceuticals and Dr. Susan VanMeter, Senior Director, Clinical Research at Mallinckrodt Pharmaceuticals for a Zoom webinar on Friday, January 22, at 12:30 – 1:30 pm. In the meeting you will hear updates of the Adrabetadex program. You can access the by clicking on the Button below:Join … Read More
Niemann-Pick Type C Caregiver Preference Survey on Benefit Risk for Potential Treatments
Dear Friends in the NPC Community, We invite families or caregivers of persons with Niemann-pick Type C disease to take a new survey that is designed to collect information about your priorities and preferences for treating Niemann-Pick Type C. It is organized by the NPC Therapy Accelerator*. In the survey, you will read about hypothetical treatments for Niemann-Pick Type C. … Read More
Positive results reported from IntraBio’s multinational clinical trial with IB1001 for treatment of Niemann-Pick disease Type C
A multinational clinical trial of IB1001 for the treatment of Niemann-Pick disease Type C (NPC) demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC. The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was … Read More
Orphazyme announces U.S. FDA acceptance and Priority Review of New Drug Application for arimoclomol for Niemann-Pick disease Type C
Today, Orphazyme announced that the U.S. Food and Drug Administration (FDA) has accepted, with Priority Review, the company’s New Drug Application (NDA) for arimoclomol for the treatment of NPC disease. The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions. This is … Read More