Niemann-Pick Disease Type C (NP-C)

Niemann-Pick Disease Type C (NP-C) It is a progressive, irreversible and chronically debilitating neuroviscerallysosomalLipid Storage Disease, which is characterized by the visceral, and neurological manifestations. 01 NP-C Classification includes NiemannPick Type C1 and Niemann-Pick Disease Type C2. Mutation in NP-C1 gene leads to Type C1 whereas mutation in NP-C2 gene leads to type C2. 02 NP-C Symptoms include sudden muscle problems like seizures, clumsiness, tremors, problems walking, sudden falls, slurred speech and trouble in moving eyes up and down. 03 It involves unique abnormalities of intracellular transport of endocytosed cholesterol with sequestration of unesterified cholesterol in lysosomes and late endosomes. 04 • DelveInsight © 2019 | All rights reserved

NP-C Epidemiology • NP-C diagnosis rate in the 7MM* is assessed to be around 50% and is expected to increase. NP-C higher prevalence was 1011 cases in 2017 in the US, followed by EU5 and Japan. • NP-C total prevalent population in the 7 Major Markets (7MM)* was • 2115 cases in 2017. • NP-C prevalence in Western Europe was approximately 1/120,000 to 1/150,000 living births. Niemann-Pick Disease Type C (NP-C) prevalence is similar in both males and females. * 7MM- USA, EU-5(Germany, Spain, Italy, France and UK), Japan • DelveInsight © 2019 | All rights reserved

NP-C Market Outlook Niemann-Pick Disease Type C therapeutic market in 7MM is expected to reach USD 162.30 million by 2028. Subtype Specific NP-C Prevalence in the United States. 5% 32% Mutations in NP-C1 gene were observed in majority of the patients whereas mutations in NP-C2 gene were reported in nearly 5% of the patient population In 2016. The United States accounts for 32% of the total sales in 2017. The United States accounts for the largest NP-C market size, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan. • DelveInsight © 2019 | All rights reserved

NP-C Market Outlook Growth of NP-C market could also increase due to the introduction of diagnostic equipment. (e.g. Biomarkers such as oxysterols help in improving NP-C diagnosis with unclear biochemical phenotypes and NP-C gene mutations) Increasing healthcare expenditure, rise in R&D activities, awareness among patients about NP-C treatment options supports NP-C market growth. To overcome toxicity, severe side effects and complications of NP-C current treatment methods, many companies are developing potential therapies for NP-C treatment. NP-Cmarket size shall experience a positive growth during the forecast period (2018-2027) • DelveInsight © 2019 | All rights reserved

NP-C Emerging Drugs and Companies Drug- VTS-270 Company- Mallinckrodt Drug- Trappsol Cyclo Company- CTD Holdings Drug- Arimoclomol Company- Orphazyme Company- IntraBio • DelveInsight © 2019 | All rights reserved

Complete Information For complete information on Niemann-Pick Disease Type C (NP-C) Market Insights, Epidemiology & Market Forecast-2028 please click the link below CLICK HERE OR Write Us At info@delveinsight.com • DelveInsight © 2019 | All rights reserved • DelveInsight © 2019 | All rights reserved

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Niemann-Pick Disease Type C (NP-C)