On February 13, 2018, Mallinckrodt Pharmaceuticals acquired Sucampo Pharmaceuticals, including VTS-270, an investigational treatment for a devastating, rare neurodegenerative and ultimately fatal disease called Niemann-Pick disease type C1 (NPC).
NPC is an inherited lysosomal storage disease caused by genetic mutations that affect cellular metabolism and can lead to the accumulation of cholesterol and lipids in the spleen, liver, lungs, bone marrow and brain. NPC affects an estimated one in approximately 100,000 live births.[i] Symptoms, which can include severe neurologic, systemic and/or psychiatric symptoms, can start at any age and are progressive.[ii] The average age for diagnosis is age 10, and most of those affected with NPC die before age 20. [iii],[iv]
Current approaches to managing NPC have not shown efficacy in significantly delaying the progression of NPC.[v]
Mallinckrodt Pharmaceuticals recognizes the extreme need for therapeutic options for NPC and is committed to advancing VTS-270 through clinical investigation for patients living with the neurologic manifestations of NPC.
VTS-270 was granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA), and orphan drug status by the FDA and European Medicines Agency (EMA), which equates to accelerated regulatory review in the U.S. and Europe. Currently, VTS-270 is being evaluated in an ongoing multi-center, randomized, double-blind Phase 2b/3 efficacy and safety study in NPC patients.
Considering the complexity of diagnosing and treating NPC, the development of VTS-270 provides Mallinckrodt with an important opportunity to help support NPC families and fulfill an essential part of its long-standing corporate mission – to manage complexity and improve lives. With that focus, Mallinckrodt remains steadfast in its efforts to advance VTS-270 through clinical development and the regulatory review and approval process.
[i] Benussi, A. Cotelli, M. et al. Preliminary Results on Long-Term Potentiation-Like Cortical Plasticity and Cholinergic Dysfunction After Miglustat Treatment in Niemann-Pick Disease Type C. JIMD Reports: Springer-Verlag Berlin Heidelberg. 2017: 19-27.
[ii] Mengel, E. et al. Niemann-Pick disease type C symptomatology: an expert-based clinical description. Orphanet Journal of Rare Diseases. 2013; 8: 166. https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-8-166.
[iii] Niemann-Pick Type C. http://www.npcfund.org/niemann-pick-type-c/.
[iv] Niemann-Pick Disease Overview – Types A, B and C. https://nnpdf.org/overview/#NPC.
[v] Patterson, M. Vecchio, D, et al. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurology. 2007; 6: 765-72.