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

I want to share an update on our adrabetadex program. In late April, Mallinckrodt received a general advice letter from the FDA regarding the adrabetadex data package submitted in support of a future regulatory filing. The agency indicated that additional data from our ongoing studies is not sufficient to demonstrate the efficacy of the drug and that an additional study … Read More

The Ara Parseghian Medical Research Fund at Notre Dame is pleased to announce the support of 17 grants for the 2020 Grant funding program. Thank you to our generous donors who continue to support our efforts to find a cure for Niemann-Pick Type C disease.  Grants funded this year will support a collaborative group of leading NPC scientists from around … Read More

We are now several months into the COVID-19 pandemic, and the Orphazyme team in the United States (U.S.) has been adjusting, just like the millions of American families seeking to maintain normal activities. We recognize the unique burden the public health crisis places on families managing treatment of rare conditions.  In January, we announced an Early Access Program (EAP) in … Read More

March 2020 A message from Orphazyme to the global community of patients, family members and healthcare providers impacted by the corona virus pandemic (COVID-19): This is an uncertain and difficult time for us all. As we face these challenges, Orphazyme’s top priority is the health and safety of patients and our employees. We have taken responsible measures, in line with … Read More

Mallinckrodt issued a press release on February 25 regarding the companies future, current settlement, and continuation of developing novel therapies and improving the lives of patients. For additional information and a link to the official press release, please visit www.advancingmnk.com  Highlights (modified from a document provided by Mallinckrodt) Mallinckrodt reached agreement in principle on terms of global settlement that would … Read More

On Friday, October 11, 2019, friends of the APMRF gathered on Notre Dame’s campus to spend time together, and learn about the Parseghian’s fight to find a cure for Niemann-Pick Type C (NPC) disease. The Parseghian’s were also joined by Cheryl and Paul Marcogliese and their two boys, Daniel and James. Special thank you to Cheryl who talked to the … Read More

Dear Toni and Niemann-Pick UK Members, Good news!  We are pleased to inform you that Mallinckrodt has confirmed with the Medicines and Healthcare Products Regulatory Agency (MHRA) that the temporary halt of Study VTS301 Part C has been lifted and the study may resume in the United Kingdom.  Mallinckrodt is working with the MHRA and the study investigators to ensure … Read More

As we continue to receive questions about the recent suspension of the open label extension portion (Part C) of our Phase 2b/3 study in the UK and France, I wanted to reach out to share another brief update based on the information we have available at this time. We understand that there may be a desire for some study participants … Read More