C型ニーマン・ピック病 (NPC) の世界市場 (2025年～2033年)

世界のC型ニーマン・ピック病 (NPC) 市場は、2024年に900万米ドルに達し、2033年には9億2,357万米ドルに達し、2025年～2033年の予測期間中に28.9％のCAGRで成長すると予測されています。

ニーマン・ピック病は、スフィンゴミエリンからセラミドとホスホコリンへの加水分解に重要な役割を果たす酸性スフィンゴミエリナーゼ酵素の欠損を特徴とするライソゾーム貯蔵病（LSD）の希少な種類です。この酵素欠損の結果、細胞膜機能に重要な役割を果たすスフィンゴミエリンが、脾臓、肝臓、肺、脳など様々な臓器に存在するマクロファージのリソソームに蓄積します。この蓄積により、脾腫、肝腫大、細胞減少、肺疾患、神経症状が生じます。

ニーマン・ピック病は、特にA型、B型、C型の3つのタイプに分類されます。現在、C型の治療薬として2つの薬剤が承認されており、その他の種類に対する先進的な治療法の実現に向けて研究が進められています。

市場力学：

促進要因と抑制要因

新製品の承認と開発活動の増加

C型ニーマン・ピック病 (NPC) は、ライソゾーム貯蔵病の中でも最も稀で致死的な疾患の一つです。NPCの発症率は、出生10万人に1人から出生13万人に1人です。2024年上半期まで、NPCに対する特定の治療法は承認されておらず、疾患管理は完全に対症療法に集中していました。しかし、2024年下半期には2つの治療薬が承認され、NPC治療の状況は一変しました。これらの医薬品は世界中の様々な市場に浸透し、診断された人々の間で高いアンメットニーズに対応することが期待されています。

例えば、米国食品医薬品局は2024年9月20日、Zevra Therapeutics, Inc.が製造するアリモクロモール（商品名MIPLYFFA）を2歳以上の成人および小児NPC患者の治療薬として承認しました。本治療薬は、NPCの神経学的症状の治療薬としてMiglustatとの併用が適応となります。Zevraの発表によると、米国と欧州には1800人近いNPC患者が存在し、米国では300人が診断されています。米国では承認後1ヵ月以内に90の処方が登録され、ゼブラ社は欧州でも早期アクセスプログラム（EAP）を通じて患者アクセスを拡大しており、このプログラムを通じて70～80人の患者を獲得する予定です。

一方、MIPLYFFAの承認からわずかなスパンで、米国FDAは2024年9月25日にIntraBio Inc.のAQNEURSA（レバセチルロイシン）を成人および小児におけるC型ニーマン・ピック病 (NPC) の治療薬として承認しました。MIPLYFFAとは異なり、この薬剤は適応症に対する単剤療法として承認されています。これは、NPC治療におけるもう一つの重要なマイルストーンです。

これらの薬剤は最近承認されたばかりであり、世界中のいくつかの市場にはまだ浸透していません。

承認薬の高コストが市場成長を抑制する可能性。

MiplyffaとAqneursaはNPC領域における画期的な治療薬となりましたが、これらの薬剤は非常に高価であるため、特に保険が適用されない患者への採用は疑問視されています。例えば、ミプリファ・カプセル1個の価格は、処方される量にもよりますが、米国466.24米ドルから1,230.00米ドルです。これらの価格と1日の投与量を考慮すると、ミプリファ治療を受ける患者の年間費用は510,542.41米ドルから1,346,778.58米ドルになります。これは低・中所得国に住む患者にとって大きな経済的負担となります。

目次

第1章 分析手法と分析範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 新製品の承認
    • NPCの診断率の向上
  • 抑制要因
    • 承認薬の高コスト
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• 疫学
• 価格分析
• パイプライン分析
• 特許分析
• 規制分析

第6章 薬剤別

• アリモクロモール（MIPLYFFA）
• レバアセチルロイシン（AQNEURSA）
• ミグルスタット

第7章 患者の種類別

• 成人
• 小児

第8章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州地域
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第9章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第10章 企業プロファイル

• Zevra Therapeutics.
  • 会社概要
  • 製品ポートフォリオ
    • 製品説明
    • 製品の主要業績評価指標（KPI）
    • 製品販売の実績・予測
    • 製品販売量
  • 財務概要
    • 企業の収益
    • 収益シェア：地域別
    • 収益予測
  • 主な発展
    • 企業合併・買収 (M&A)
    • 主な製品開発活動
    • 規制当局の承認等
  • SWOT分析
• IntraBio.
• Johnson & Johnson Services, Inc.
• Cyclo Therapeutics, Inc
• Azafaros B.V.
• 各市場参入企業に対しても同様のデータが提供されます。

第11章 付録

The global Niemann-Pick Disease Type C (NPC) market reached US$ 9.00 million in 2024 and is expected to reach US$ 923.57 million by 2033, growing at a CAGR of 28.9% during the forecast period of 2025-2033.

Niemann-Pick Disease is a rare type of lysosomal storage disease (LSD) characterized by the deficiency of the acid sphingomyelinase enzyme that plays a crucial role in the hydrolysis of sphingomyelin to ceramide and phosphocholine. As a result of this enzyme deficiency, sphingomyelin, which plays a crucial role in cell membrane function, accumulates in the lysosomes of macrophages present across various organs such as the spleen, liver, lungs, and brain. This accumulation results in splenomegaly, hepatomegaly, cytopenia, lung diseases, and neurological manifestations.

Niemann-Pick Disease is classified into 3 types, notably type A, type B, and type C. At present, two drugs have been approved for the treatment of type C disease, and research is ongoing to bring advanced therapies for the other types.

Market Dynamics: Drivers & Restraints

Rising Novel Product Approvals and Development Activities

Niemann-Pick Disease Type C (NPC) is one of the rarest and most fatal types of lysosomal storage diseases. The incidence of NPC ranges from 1 in 100,000 live births to 1 in 130,000 live births. Up until the 1H of 2024, no specific therapies were approved for NPC, and disease management completely focused on symptomatic relief. However, in the 2H of 2024, two drugs were approved for the indication, which has revolutionized the NPC treatment landscape. These drugs are expected to penetrate various markets around the globe and are anticipated to address the high unmet needs among the diagnosed population.

For instance, on September 20, 2024, the U.S. Food and Drugs Administration approved arimoclomol with the brand name MIPLYFFA, manufactured by Zevra Therapeutics, Inc., for the treatment of adult and pediatric NPC patients above 2 years of age. The drug is indicated in combination with Miglustat for the treatment of neurological manifestations of NPC. As per Zevra, nearly 1800 NPC patients were present in the U.S. and Europe, with 300 diagnosed in the United States. Within a month of approval, 90 prescriptions were enrolled in the U.S., and Zevra is extending patient access in Europe through Early Access Programs (EAP) and is planning to reach 70 to 80 patients through this program.

On the other hand, within a short span of MIPLYFFA approval, the U.S. FDA also approved IntraBio Inc's AQNEURSA (levacetylleucine) on September 25, 2024, for the treatment of Niemann-Pick Disease Type C (NPC) in the adult and pediatric population. Unlike MIPLYFFA, this drug is approved as monotherapy for the indication. This is another key milestone in the NPC treatment landscape.

These drugs are only recently approved, and they are yet to penetrate several markets across the globe, which is anticipated to happen in the forecast period, driving the overall market growth.

High- Cost of Approved Drugs May Restrain the Market Growth.

Miplyffa and Aqneursa have become revolutionary therapies in the NPC landscape, however, these drugs are highly expensive, which makes their adoption questionable, especially in patients with no insurance coverage. For instance, the cost of 1 Miplyffa capsule can range between US$ 466.24 to US$ 1,230.00 depending on the dosage prescribed. Considering these prices and a daily dosage, the annual cost of patients on Miplyffa treatment can range between US$ 510,542.41 to US$ 1,346,778.58. This can be a huge economic burden to patients living in low-to-middle-income countries.

Segment Analysis

The global Niemann-Pick Disease Type C (NPC) market is segmented based on drug, patient type, and region.

Arimoclomol (MIPLYFFA) in the drug segment is dominating the market.

Arimoclomol, sold under the brand name MIPLYFFA and manufactured by Zevra Therapeutics, is the first prescription medication approved for the treatment of Niemann-Pick Disease Type C (NPC). The drug is recommended for use in adult and pediatric patients above 2 years of age in combination with miglustat and is given at various doses three times a day based on the patient's actual body weight. MIPLYFFA received marketing authorization from the U.S. FDA on September 20, 2024, and was commercially launched in the U.S. market on November 21, 2024. This drug has also received the orphan medicinal product designation from the European Commission, however, the approval is still pending. Although not approved in the European Market, Zevra is providing MIPYFFA to patients in Europe through an early access program. This reflects Zevra's strong efforts to commercialize its products globally, giving it a competitive edge in the market. Zevra's R&D, manufacturing, and investment capabilities further drive the higher sales gain for MIPLYFFA. As per DataM intelligence, MIPLYFFA is expected to gain a sales value of ~ 350 million by 2030.

Geographical Analysis

North America dominated the Niemann-Pick Disease Type C (NPC) market.

The factors contributing to the region's dominance include high diagnosis and treatment rates and the first availability of approved drugs.

As per Zevra Therapeutics Inc., the estimated population in the U.S. with NPC is approximately 900, of which 300 patients are currently diagnosed. This represents a diagnosis rate of ~33% in the country, which is higher compared to other countries of the world. In combination with the high diagnosis rate, the revolutionary treatments Miplyffa and Aqneursa were first approved in the U.S. market. It means that the patient population in the country receives first access to these drugs, and the manufacturers sell them at higher prices and generate most of their revenue from the country. This is a major contributing factor to the North America market dominance.

In addition, the favorable yet stringent regulatory scenario in the U.S. and their involvement in supporting drug discovery in development is a key factor for manufacturers preferring the U.S. for their product development, launch, and commercialization. The U.S. FDA provides special designations to drugs that can accelerate their approval time and provide early market access. For instance, apart from Miplyffa and Aqneursa, the U.S. FDA has granted Orphan Drug Designation to Cyclo Therapeutics, Inc.'s Trappsol Cyclo, which is currently in phase III trials for NPC. This designation can enable the drug to undergo a priority review process and receive accelerated approval. Once this is approved, the country to get access is the U.S.

Hence, considering all the factors, the North American region is designated as a dominant region in the global Niemann-Pick Disease Type C (NPC) market.

Competitive Landscape

The major players in the Niemann-Pick Disease Type C (NPC) market are Zevra Therapeutics, IntraBio, and Johnson & Johnson Services, Inc., among others.

Key Developments

• On February 7, 2025, Cyclo Therapeutics, Inc. announced the positive preliminary data from its ongoing phase III trial of Trappsol Cyclo, which is designated for the treatment of Niemann-Pick Disease Type C (NPC)1 (NPC1). In 7 among 8 subject populations, Trappsol Cyclo has stabilized and improved the Clinical Global Impression - Severity (CGI-S) scale at 24 weeks, and CGI-S stabilization and improvement was seen in 6 among 7 subjects who received treatment for 48 weeks. The company anticipates interim 48-week data in H1 of 2025, after which regulatory submissions will be done for marketing authorization.
• On July 16, 2024, Azafaros B.V. announced positive clinical trial results of the phase II trial evaluating nizubaglustat for GM2 gangliosidosis or Niemann-Pick Disease Type C (NPC). The study has shown a positive safety profile of nizubaglustat among the subject population, and upon this success, Azafaros plans to continue pursuing the drug in phase II trials, which are anticipated to begin in 2025.

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials and product pipelines and forecasts upcoming pharmaceutical advancements.
• Type Performance & Market Positioning: Analyze product performance, market positioning, and growth potential to optimize strategies.
• Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: This covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyze competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient Type delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance product safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global melanoma therapeutics market report would provide approximately 45 tables, 46 figures, and 180 pages.

Target Audience 2024

• Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Drug
• 3.2. Snippet by Patient Type
• 3.3. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. New Product Approvals
    • 4.1.1.2. Improved Diagnosis Rate of NPC
  • 4.1.2. Restraints
    • 4.1.2.1. High Cost of Approved Drugs
  • 4.1.3. Opportunities
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Forces Analysis
• 5.2. Epidemiology
• 5.3. Pricing Analysis
• 5.4. Pipeline Analysis
• 5.5. Patent Analysis
• 5.6. Regulatory Analysis

6. By Drug

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 6.1.2. Market Attractiveness Index, By Drug
• 6.2. Arimoclomol (MIPLYFFA)*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Levacetylleucine (AQNEURSA)
• 6.4. Miglustat

7. By Patient Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type
  • 7.1.2. Market Attractiveness Index By Patient Type
• 7.2. Adult*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Pediatric

8. By Region

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 8.1.2. Market Attractiveness Index, By Region
• 8.2. North America
  • 8.2.1. Introduction
  • 8.2.2. Key Region-Specific Dynamics
  • 8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type
  • 8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.2.5.1. U.S.
    • 8.2.5.2. Canada
    • 8.2.5.3. Mexico
• 8.3. Europe
  • 8.3.1. Introduction
  • 8.3.2. Key Region-Specific Dynamics
  • 8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type
  • 8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.3.5.1. Germany
    • 8.3.5.2. U.K.
    • 8.3.5.3. France
    • 8.3.5.4. Spain
    • 8.3.5.5. Italy
    • 8.3.5.6. Rest of Europe
• 8.4. South America
  • 8.4.1. Introduction
  • 8.4.2. Key Region-Specific Dynamics
  • 8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type
  • 8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.4.5.1. Brazil
    • 8.4.5.2. Argentina
    • 8.4.5.3. Rest of South America
• 8.5. Asia-Pacific
  • 8.5.1. Introduction
  • 8.5.2. Key Region-Specific Dynamics
  • 8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type
  • 8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.5.5.1. China
    • 8.5.5.2. India
    • 8.5.5.3. Japan
    • 8.5.5.4. South Korea
    • 8.5.5.5. Rest of Asia-Pacific
• 8.6. Middle East and Africa
  • 8.6.1. Introduction
  • 8.6.2. Key Region-Specific Dynamics
  • 8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
  • 8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Patient Type

9. Competitive Landscape

• 9.1. Competitive Scenario
• 9.2. Market Positioning/Share Analysis
• 9.3. Mergers and Acquisitions Analysis

10. Company Profiles

Key Market Players

• 10.1. Zevra Therapeutics.*
  • 10.1.1. Company Overview
  • 10.1.2. Product Portfolio
    • 10.1.2.1. Product Description
    • 10.1.2.2. Product Key Performance Indicators (KPIs)
    • 10.1.2.3. Historic and Forecasted Product Sales
    • 10.1.2.4. Product Sales Volume
  • 10.1.3. Financial Overview
    • 10.1.3.1. Company Revenue's
    • 10.1.3.2. Geographical Revenue Shares
    • 10.1.3.3. Revenue Forecasts
  • 10.1.4. Key Developments
    • 10.1.4.1. Mergers & Acquisitions
    • 10.1.4.2. Key Product Development Activities
    • 10.1.4.3. Regulatory Approvals, etc.
  • 10.1.5. SWOT Analysis
• 10.2. IntraBio.
• 10.3. Johnson & Johnson Services, Inc.

Emerging Market Players

• 10.4. Cyclo Therapeutics, Inc
• 10.5. Azafaros B.V.
• Similar data will be provided for each market player.

LIST NOT EXHAUSTIVE

11. Appendix

• 11.1. About Us and Services
• 11.2. Contact Us