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시장보고서
상품코드
1983233
니만피크병 C형(NPC) 치료 시장 : 약물 유형별, 적응증별, 유통 채널별, 지역별Niemann-Pick Disease Drug Type C Treatment Market, By Drug Type, By Indication, By Distribution Channel, By Geography |
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니만피크병 C형(NPC) 치료 시장은 2026년에 1억 90만 달러로 추정되며, 2033년까지 4억 600만 달러에 달할 것으로 예상됩니다. 2026년부터 2033년까지 CAGR 22%로 성장할 것으로 전망됩니다.
| 보고서 범위 | 보고서 상세 | ||
|---|---|---|---|
| 기준 연도: | 2025년 | 2026년 시장 규모: | 1억 90만 달러 |
| 과거 데이터 기간: | 2020-2024년 | 예측 기간: | 2026-2033년 |
| 2026년부터 2033년까지 예측 기간 CAGR: | 22.00% | 2033년 시장 규모 예측: | 4억 600만 달러 |
니만피크병 C형(NPC)은 희귀한 유전성 질환입니다. 이 유형의 유전자 변이로 인해 콜레스테롤과 기타 지질이 간, 비장 또는 폐에 축적됩니다. 또한, 뇌에도 영향을 미칩니다. NPC의 증상 발현은 연령에 따라 다릅니다. 이들은 유아형(출생 후부터 6세 미만), 청소년형(6세-15세), 성인형(15세 이상)으로 분류됩니다. 영아에서 NPC는 α-1-항트립신 결핍증, 티로신혈증, 고셔병, 특발성 신생아 담즙 정체, 각종 선천성 감염 등의 질환을 유발합니다.
시장 역학
희귀 질환에 대한 정부의 노력이 증가함에 따라 예측 기간 동안 세계 니만피크병 C형(NPC) 치료 시장의 성장이 촉진될 것으로 예상됩니다. 예를 들어, 2021년 11월 이탈리아 상원은 만장일치로 희귀질환 관련 통합 법안을 통과시켰습니다. 이 법안의 목적은 이탈리아 전역에서 희귀질환의 치료를 통일하고 연구의 진전을 촉진하는 것입니다. 또한, 이 법안에는 희귀질환 환자를 위한 연대기금 설립도 규정되어 있으며, 2022년부터 연간 101만 달러의 초기 예산이 책정되어 있습니다.
시장 관계자들의 제휴 및 계약 증가는 예측 기간 동안 세계 니만피크병 C형(NPC) 치료 시장의 성장을 견인할 것으로 예상됩니다. 예를 들어, 2022년 5월 전문 제약사 Kempharm, Inc.는 세계 바이오 제약사 ORPHAZYME A/S와 니만피크병 C형(NPC) 치료제로 개발 중인 경구 투여형 퍼스트 인 클래스 열충격단백질(HSP) 증폭제인 알리모크로몰을 인수하기로 합의했다고 발표했습니다.
시장 진입 기업들의 활발한 연구개발 활동이 예측 기간 동안 시장 성장을 견인할 것으로 예상됩니다. 예를 들어, 2021년 6월, 임상 단계의 생명공학 기업 Cyclo Therapeutics, Inc.는 NPC1 치료를 목적으로 하는 하이드록시프로필 β-시클로덱스트린의 독자적인 제제인 Trappsol Cyclo를 평가하기 위한 임상 3상 주요 시험(TransportNPC)의 시작을 발표했습니다. TransportNPC 시험은 NPC1 환자를 대상으로 Trappsol Cyclo 2,000 mg/kg과 표준치료(SOC)의 안전성, 내약성, 유효성을 위약 및 SOC와 비교 평가하기 위해 설계된 무작위 배정, 이중맹검, 위약 대조, 병렬군 간, 다기관 공동 시험입니다.
본 시험의 주요 특징.
- 이 보고서는 2021년을 기준 연도로 하여 세계 니만피크병 C형(NPC) 치료 시장과 예측 기간(2022-2030년)의 시장 규모에 대한 상세한 분석을 제공합니다.
- 이 보고서는 각 부문의 잠재적 수익 기회를 밝히고, 이 시장의 매력적인 투자 제안 매트릭스를 설명합니다.
- 또한 시장 촉진요인, 억제요인, 기회, 신제품 출시 및 승인, 지역별 전망, 주요 기업의 경쟁 전략에 대한 중요한 인사이트도 제공합니다.
- 이 보고서는 다음과 같은 매개 변수를 기반으로 세계 니만피크병 C형(NPC) 치료 시장의 주요 기업 프로파일을 작성했습니다. 구체적으로 기업 개요, 재무 실적, 제품 포트폴리오, 지리적 분포, 유통 전략, 주요 개발 및 전략, 향후 계획 등입니다.
- 이 보고서의 내용을 통해 기업의 마케팅 담당자와 경영진은 향후 제품 출시, 기술 업그레이드, 시장 확대 및 마케팅 전략에 대한 정보에 입각한 의사결정을 내릴 수 있을 것입니다.
- 이 보고서는 투자자, 공급업체, 제조업체, 유통업체, 신규 진입자, 금융 분석가 등 업계의 다양한 이해관계자를 대상으로 합니다.
- 이해관계자들은 세계 니만피크병 C형(NPC) 치료 시장 분석에 사용된 다양한 전략 매트릭스를 활용하여 의사결정을 원활하게 내릴 수 있을 것입니다.
목차
제1장 조사 목적과 가정
제2장 시장 개요
제3장 시장 역학, 규제 및 동향 분석
제4장 세계의 니만피크병 C형(NPC) 치료 시장 : 약물 유형별, 2026-2033년
제5장 세계의 니만피크병 C형(NPC) 치료 시장 : 적응증별, 2026-2033년
제6장 세계의 니만피크병 C형(NPC) 치료 시장 : 유통 채널별, 2026-2033년
제7장 세계의 니만피크병 C형(NPC) 치료 시장 : 지역별, 2026-2033년
제8장 경쟁 구도
제9장 섹션
KSM 26.04.20Niemann-Pick Disease Drug Type C Treatment Market is estimated to be valued at USD 100.9 Mn in 2026 and is expected to reach USD 406.0 Mn by 2033, growing at a compound annual growth rate (CAGR) of 22% from 2026 to 2033.
| Report Coverage | Report Details | ||
|---|---|---|---|
| Base Year: | 2025 | Market Size in 2026: | USD 100.9 Mn |
| Historical Data for: | 2020 To 2024 | Forecast Period: | 2026 To 2033 |
| Forecast Period 2026 to 2033 CAGR: | 22.00% | 2033 Value Projection: | USD 406.0 Mn |
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
- 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 Region
- North America
- U.S.
- Canada
- Latin America
- Brazil
- Argentina
- Mexico
- Rest of Latin America
- Europe
- Germany
- U.K.
- Spain
- France
- Italy
- Russia
- Rest of Europe
- Asia Pacific
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
- Middle East & Africa
- GCC Countries
- Israel
- Rest of Middle East & Africa
- Global Niemann-Pick Disease Drug Type C Treatment Market - Competitive Landscape
- Johnson & Johnson
- Intrabio
- Cyclo Therapeutics, Inc.
- Kempharm, Inc.
- Azafaros B.V.
- StrideBio
- Sarepta Therapeutics, Inc.
Table of Contents
1. Research Objectives and Assumptions
- Research Objectives
- Assumptions
- Abbreviations
2. Market Overview
- Report Description
- Market Definition and Scope
- Executive Summary
- Market Snippet, By Drug Type
- Market Snippet, By Indication
- Market Snippet, By Distribution Channel
- Market Snippet, By Region
- Coherent Opportunity Map (COM)
3. Market Dynamics, Regulations, and Trends Analysis
- Market Dynamics
- Drivers
- Restraints
- Market Opportunities
- Impact Analysis
- Porter's Analysis
- Industry Trends
- Pipeline Analysis
- Key Highlights
- Epidemiology
- Reimbursement Scenario
- Regulatory Scenario
- Product Launch and Approvals
- Acquisition and Collaboration Scenario
4. Global Niemann-Pick Disease Drug Type C Treatment Market, By Drug Type, 2026-2033 (US$ Million)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2026-2033
- Segment Trends
- Phase III Drug
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million)
- Trappsol Cyclo
- IB1001
- Marketed Drug
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million)
- Miglustat (Zavesca)
5. Global Niemann-Pick Disease Drug Type C Treatment Market, By Indication, 2026-2033 (US$ Million)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2026-2033
- Segment Trends
- Niemann-Pick Disease Drug Type C1
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million)
- 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)
- Introduction
- Market Share Analysis, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, 2026-2033
- Segment Trends
- Hospitals Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million)
- Online Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2026-2033, (US$ Million)
- 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)
- Introduction
- Market Share Analysis, By Region, 2026 and 2033 (%)
- Y-o-Y Growth Analysis, For Regions, 2026-2033
- Regional Trends
- 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)
- U.S.
- Canada
- 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)
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- 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)
- Germany
- U.K.
- France
- Italy
- Spain
- Russia
- Rest of Europe
- 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)
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
- 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)
- GCC
- Israel
- Rest of Middle East
- 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)
- South Africa
- Central Africa
- North Africa
8. Competitive Landscape
- Company Profiles
- Johnson & Johnson
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Intrabio
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Cyclo Therapeutics, Inc.
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Kempharm, Inc
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Azafaros B.V
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- StrideBio
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Sarepta Therapeutics, Inc.
- Company Overview
- Product Portfolio
- Financial Performance
- Recent Developments/Updates
- Strategies
- Johnson & Johnson
9. Section
- Research Methodology
- Analyst Views
- About us
- Contact
