Mallinckrodt Committed to Niemann-Pick Disease Type C1 Patients, Therapy

Mallinckrodt Pharmaceuticals recognizes the extreme need for therapeutic options for Niemann-Pick disease type C1 (NPC), a devastating, rare neurodegenerative disease. Building on our established position in developing treatments for rare diseases, in February 2018, we acquired Sucampo Pharmaceuticals and VTS-270. We are committed to advancing it through clinical investigation.

Currently, VTS-270 is being evaluated in an ongoing Phase 2b/3 efficacy and safety study in NPC patients. Our Mallinckrodt clinical team is diligently overseeing this multi-center, randomized, double-blind trial. 

Considering the complexity of diagnosing and treating NPC, the development of VTS-270 provides us with an important opportunity to fulfill an essential part of our long-standing corporate mission – to manage complexity and improve lives. With that focus, we remain steadfast in our efforts to advance VTS-270 through clinical development and the regulatory review and approval process.

The commitment required to participate in a clinical trial is substantial, and we want to recognize and thank the patients, families, advocates and healthcare providers who are involved. Without you, it would not be possible to advance our understanding of NPC.

We appreciate that there are many questions from the community regarding the status of VTS-270 and we are committed to providing updates as appropriate.  As such, we are engaged in regular dialogue with the national and international Niemann-Pick disease advocacy community.  And, we will continue to seek to understand what is needed by listening and partnering with the community.  We are in this together.  

Thank you for your interest and continued support of the VTS-270 clinical development program. Please do not hesitate to contact us if you have any questions at

Derek Naten on behalf of the Mallinckrodt VTS-270 team
Sr. Director, Government Affairs & Advocacy Relations
Mallinckrodt Pharmaceuticals