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Read here market analysis report on NIEMANN-PICK DISEASE TYPE C TREATMENT
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NIEMANN-PICK DISEASE TYPE C TREATMENT MARKET ANALYSIS Niemann-Pick disease type C (NPC) is a rare genetic disorder that is a type of lysosomal storage disorder. NPC leads to accumulation of cholesterol and other fatty substances in different tissues of the body, such as brain and others. The onset of specific symptoms for NPC varies from person to person. In most cases, the symptoms appear in the childhood, which progresses to cause life-threatening complications with increasing age. NPC occurs as a result of mutations in the NPC 1 gene and NPC 2 gene, which is inherited in an autosomal recessive manner.
Market Dynamics Market players are engaged in receiving approvals from regulatory authorities, which is one of the important steps for launching products in the market. This is expected to drive growth of the global Niemann-Pick disease type C treatment market over the forecast period. For instance, on September 16, 2020, Orphazyme A/S, a biopharmaceutical company, received the U.S. Food and Drug Administration (FDA) approval, with priority review for New Drug Application (NDA), for arimoclomol for the treatment of NPC.
Market players are involved in conducting research and development in order to identify targets for the disease, against which a new treatment can be developed in the future. For instance, in 2018, Centogene AG Rostock, a genetic diagnostic company, initiated a clinical trial to prepare a cell culture from patients suffering from Niemann Pick disease type C for the identification of novel proteins involved in pathways that leads to disease progression. This will also aid in early diagnosis. The proteins can be targeted for the development of treatment options for NPC, which is expected to drive growth of the market.
Key features of the study: • This report provides an in-depth analysis of global Niemann-Pick disease type C treatment market, provides market size (US$ Million), and compound annual growth rate (CAGR %) for the forecast period (2020 – 2027), considering 2019 as the base year • It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrix for this market • This study also provides key insights about market drivers,
restraints, opportunities, new product launches or approval, regional outlook, and competitive strategies adopted by the leading players • It profiles leading players in the global Niemann-Pick disease type C treatment market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies, and future plans • Key companies covered as a part of this study include OrphazymeApS, CTD Holdings Inc, Neurotrope Inc, Okklo Life Sciences Bv, Intrabio Ltd, Azafaros, Centogene AG Rostock, Actelion Pharmaceuticals Ltd, Mallinckrodt Pharmaceuticals, Merck & Co., Inc., Alexion Pharmaceuticals, Sanofi Genzyme, and Aldagen Inc.
Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launch, technology up-gradation, market expansion, and marketing tactics • The global Niemann-Pick disease type C treatment market report caters to various stakeholders in this industry including investors, suppliers, manufacturers, distributors, new entrants, and financial analysts • Stakeholders would have ease in decision making through various strategy matrices used in analyzing the Niemann-Pick disease type C treatment market
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Figure 1: Global Niemann-Pick Disease Type C Treatment Market Share (%) Analysis, By Disease Type, 2020
Figure 2: Global Niemann-Pick Disease Type C Treatment Market Value (US$ Mn), by Region, 2020
Company Profiles Major players operating in the global Niemann-Pick diseases type C treatment market include OrphazymeApS, CTD Holdings Inc, Neurotrope Inc, Okklo Life Sciences Bv, Intrabio Ltd, Azafaros, Centogene AG Rostock, Actelion Pharmaceuticals Ltd, Mallinckrodt Pharmaceuticals, Merck & Co., Inc., Alexion Pharmaceuticals, Sanofi Genzyme, and Aldagen Inc.
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