Niemann-Pick Disease is a type of lipid storage disorder characterised by accumulation of fatty substances in organs such as spleen, liver, and brain, eventually causing cell death resulting in malfunctioning of major organ systems. Its pathogenesis involves deficiency of a lysosomal enzyme called acid sphingomyelinase, and is mainly caused by genetic mutation. Niemann-Pick disease is majorly prevalent in North America, South America, Europe, Africa, Asia, and Australia. There are three main types of Niemann-Pick Disease called Type A and B (Acid Sphingomyelinase Deficiency) and Niemann-Pick Disease Type C (NPC). Approximately 1,200 cases of NPA and NPB have been recorded, worldwide, with the majority being Type B or the intermediate form. Cure for Type A disease is yet to be found and researchers are continuously trying new possible treatments including enzyme replacement and gene therapy. For Type D, there is no effective treatment; however, few drugs are available for curing the nervous system symptoms of Type C disease.
Growth in the pipeline of Niemann-Pick disease is anticipated over next few years on account of increasing funding and research support from various non-profit organisations, lack of effective treatment, easy orphan designation from U.S. FDA, and advancements in the technology for gene and enzyme replacement therapy.
In April 2017, Sucampo Pharmaceuticals acquired Vtesse Inc., including its clinical stage Niemann-Pick disease type C1 drug, VTS-270. However, the Vtesse team will continue the ongoing Phase IIb/III clinical trials for VTS-270. The trials are expected to readout in mid-2018 and the drug could reach the market as early as 2019. In March 2017, CTD Holdings, Inc. began recruitment for a Phase I EU clinical trials of Trappsol® cycloTM to be used for the treatment of Niemann-Pick Disease Type C. In addition to the EU study, CTD Holdings is planning to initiate Phase I clinical trials in the U.S. for evaluating intravenous administration of Trappsol® Cyclo™ in NPC patients. In June 2016, Orphazyme APS announced dosing of two patients with arimoclomol in the Phase 3 ‘AIDNPC’ Niemann-Pick Disease Type C clinical trial programme. For the study, Orphazyme is seeking approval for clinical trial applications with eight national competent authorities and ethics committees.
In June 2015, the U.S. Food and Drug Administration granted breakthrough therapy designation for Olipudase Alfa of Sanofi Genzyme for the treatment of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease Type B. In July 2013, La Jolla Pharmaceutical Company, Inc. received an orphan designation for its drug allopregnanolone, to be used in the treatment of Neimann-Pick Disease Type C. In November 2008, Zavesca® of Actelion Pharmaceuticals Ltd received an orphan designation for the treatment of Neimann-Pick Disease Type C.
The key market players having a therapeutic pipeline for Niemann-Pick disease include CTD Holdings, Inc., Sanofi Genzyme, Alexion Pharmaceuticals, Actelion Pharmaceuticals Ltd, Vtesse Inc., La Jolla Pharmaceutical Company, Merck & Co., Inc., Okklo Life Sciences BV, Orphazyme ApS and Aldagen Inc.