Niemann-Pick Disease Drug Type C Treatment Market

研究執行與發布：Coherent Market Insights　·　發布日期 2026-02-26　·　168 頁
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2026 年市場規模: USD 100.9 Mn
2033 年預估規模: USD 406.0 Mn
年複合成長率 CAGR: 22%

以上數字由 Coherent Market Insights 於本報告中提出，量子訊息原樣引用。

出版商 Coherent Market Insights 產業別 Healthcare & Life Sciences出版日期 2026-02-26頁數 168報告編號 CMI4275

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Niemann-Pick Disease Drug Type C Treatment Market, By Drug Type (Phase III Drug (Trappsol Cyclo, IB1001), Marketed Drug (Miglustat (Zavesca))), By Indication (Niemann-Pick Disease Drug Type C1, Niemann-Pick Disease Drug Type C2), By Distribution Channel (Hospital Pharmacies, Online Pharmacies, Retail Pharmacies), By Geography (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

報告摘要

Niemann-Pick Type C (NPC) is a rare inherited disease. This type of genetic mutation causes cholesterol and other fats to build up in the liver, spleen, or lungs. It has an impact on the brain as well. The onset of symptoms for NPC may begin at different ages. It has been grouped as: infantile (after birth to less than 6 years), juvenile (6 to 15 years), and adults (15 years to greater). In infants, NPC leads to disorders such as alpha-1-antitrypsin deficiency, tyrosinemia, Gaucher disease, idiopathic neonatal cholestasis, and various congenital infections. Market Dynamics Increasing government initiatives for rare diseases is expected to foster global niemann-pick disease type C treatment market growth over the forecast Period. For instance, in November 2021, the Italian senate unanimously approved the consolidated act on rare diseases. The aim of the act is to make the treatment of rare diseases uniform throughout the Italian territory and to promote progress in research. Moreover, the act also provides for the establishment of solidarity fund for people with rare diseases, with an initial budget of US$ 1.01 million per year starting from 2022. Rising numbers of collaborations and agreements by market players are expected to drive growth of the global Niemann-pick disease drug type C treatment market over the forecast period. For instance, in May 2022, Kempharm, Inc., a specialty pharmaceutical company, announced an agreement with ORPHAZYME A/S, a global biopharmaceutical company, to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC) Increasing research and development activities by market players are expected to drive growth of market over the forecast period. For instance, in June 2021, Cyclo Therapeutics, Inc., a clinical-stage biotechnology company, announced the commencement of its pivotal Phase 3 study (TransportNPC) for the evaluation of Trappsol Cyclo, a proprietary formulation of hydroxypropyl beta cyclodextrin, delivered intravenously, for treating Niemann-Pick Disease Type C1 (NPC1). The TransportNPC study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg of Trappsol Cyclo administered intravenously and standard of care (SOC) compared to placebo administered intravenously and SOC in patients with NPC1. Key features of the study • This report provides an in-depth analysis of the global Niemann-pick disease drug type C treatment market , market size (US$ Million), and compound annual growth rate (CAGR%) for the forecast period (2022-2030), considering 2021 as the base year • It elucidates potential revenue opportunities across different segments and explains an 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 drug 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 Livewise Johnson & Johnson, Intrabio, Cyclo Therapeutics, Inc., Kempharm, Inc., Azafaros B.V., StrideBio. Sarepta Therapeutics, Inc. • Insights from this report would allow marketers and the management authorities of the companies to make an informed decision regarding their future product launch, technology up-gradation, market expansion, and marketing tactics. • The global Niemann-pick disease drug 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 global Niemann-pick disease drug type C treatment market Market Segmentation • By Drug Type o Phase III Drug  Trappsol Cyclo  IB1001 o Marketed Drug  Miglustat (Zavesca) • By Indication o Niemann-Pick Disease Drug Type C1 o Niemann-Pick Disease Drug Type C2 • By Distribution Channel o Hospital Pharmacies o Online Pharmacies o Retail Pharmacies • By Region o North America  U.S.  Canada o Latin America  Brazil  Argentina  Mexico  Rest of Latin America o Europe  Germany  U.K.  Spain  France  Italy  Russia  Rest of Europe o Asia Pacific  China  India  Japan  Australia  South Korea  ASEAN  Rest of Asia Pacific o Middle East & Africa  GCC Countries  Israel  Rest of Middle East & Africa • Global Niemann-Pick Disease Drug Type C Treatment Market – Competitive Landscape o Johnson & Johnson o Intrabio o Cyclo Therapeutics, Inc. o Kempharm, Inc. o Azafaros B.V. o StrideBio o Sarepta Therapeutics, Inc.

目錄 Table of Contents

Table of Contents 1. Research Objectives and Assumptions o Research Objectives o Assumptions o Abbreviations 2. Market Overview o Report Description  Market Definition and Scope o Executive Summary  Market Snippet, By Drug Type  Market Snippet, By Indication  Market Snippet, By Distribution Channel  Market Snippet, By Region o Coherent Opportunity Map (COM) 3. Market Dynamics, Regulations, and Trends Analysis o Market Dynamics  Drivers  Restraints  Market Opportunities o Impact Analysis o Porter’s Analysis o Industry Trends o Pipeline Analysis o Key Highlights o Epidemiology o Reimbursement Scenario o Regulatory Scenario o Product Launch and Approvals o Acquisition and Collaboration Scenario 4. Global Niemann-Pick Disease Drug Type C Treatment Market, By Drug Type, 2026-2033 (US$ Million) o Introduction  Market Share Analysis, 2026 and 2033 (%)  Y-o-Y Growth Analysis, 2026-2033  Segment Trends o Phase III Drug  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) o Trappsol Cyclo o IB1001 o Marketed Drug  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) o Miglustat (Zavesca) 5. Global Niemann-Pick Disease Drug Type C Treatment Market, By Indication, 2026-2033 (US$ Million) o Introduction  Market Share Analysis, 2026 and 2033 (%)  Y-o-Y Growth Analysis, 2026-2033  Segment Trends o Niemann-Pick Disease Drug Type C1  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) o Niemann-Pick Disease Drug Type C2  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) 6. Global Niemann-Pick Disease Drug Type C Treatment Market, By Distribution Channel, 2026-2033 (US$ Million) o Introduction  Market Share Analysis, 2026 and 2033 (%)  Y-o-Y Growth Analysis, 2026-2033  Segment Trends o Hospitals Pharmacies  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) o Online Pharmacies  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) o Retail Pharmacies  Introduction  Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million) 7. Global Niemann-Pick Disease Drug Type C Treatment Market, By Region, 2026-2033 (US$ Million) o Introduction  Market Share Analysis, By Region, 2026 and 2033 (%)  Y-o-Y Growth Analysis, For Regions, 2026-2033  Regional Trends o North America  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Distribution Channels, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country, 2026-2033 (US$ Mn) o U.S. o Canada o Latin America  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Distribution Channels, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country, 2026-2033 (US$ Mn) o Brazil o Mexico o Argentina o Rest of Latin America o Europe  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Distribution Channel, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country, 2026-2033 (US$ Mn) o Germany o U.K. o France o Italy o Spain o Russia o Rest of Europe o Asia Pacific  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Distribution Channels, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country, 2026-2033 (US$ Mn) o China o India o Japan o Australia o South Korea o ASEAN o Rest of Asia Pacific o Middle East  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country, 2026-2033 (US$ Mn) o GCC o Israel o Rest of Middle East o Africa  Market Size and Forecast, By Drug Type, 2026-2033 (US$ Mn)  Market Size and Forecast, By Indication, 2026-2033 (US$ Mn)  Market Size and Forecast, By Distribution Channels, 2026-2033 (US$ Mn)  Market Size and Forecast, By Country/Region, 2026-2033 (US$ Mn) o South Africa o Central Africa o North Africa 8. Competitive Landscape o Company Profiles  Johnson & Johnson o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  Intrabio o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  Cyclo Therapeutics, Inc. o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  Kempharm, Inc o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  Azafaros B.V o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  StrideBio o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies  Sarepta Therapeutics, Inc. o Company Overview o Product Portfolio o Financial Performance o Recent Developments/Updates o Strategies 9. Section o Research Methodology o Analyst Views o About us o Contact

提及公司

Johnson & JohnsonIntrabioCyclo Therapeutics, Inc.Kempharm, Inc.Azafaros B.V.StrideBioSarepta Therapeutics, Inc.

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