ニーマンピック病C型：機会の評価と予測（更新版）

ニーマンピック病C型（NPC）の有病率は、主要3市場の人口増加に伴い安定して増加しており、AGRは0.29％です。

現在、主要3市場におけるNPCの治療レジメンは、ミグルスタット（ヤルゲサ、ザベスカまたはジェネリック）の使用に完全に限定されています。

この領域には、低毒性または最小の毒性を有する治療薬や、有効性の向上した新治療薬など、複数のアンメットニーズが存在します。

NPCの後期研究開発パイプラインは、異なる作用機序を持つ医薬品の開発を重視しています。

NPC人口の安定した増加と新治療薬の市場参入により、予測期間を通じて着実な成長が見込まれます。

当レポートでは、ニーマンピック病C型（NPC）の主要3市場（米国、ドイツ、英国）について調査分析し、NPCの概要、市場収益の予測、パイプラインの分析、現在と将来の市場競合の分析などを提供しています。

目次

第1章 エグゼクティブサマリー

• エグゼクティブサマリー

第2章 疾患の概要

• ニーマンピック病C型（NPC）の概要
• NPCのSWOT分析
• NPCの分類

第3章 疫学

• ニーマンピック病C型の有病率（男女、2021年～2031年）
• 情報源と調査手法

第4章 現在の治療オプション

• 治療パラダイム
• 上市済みの製品
• 製品プロファイル

第5章 アンメットニーズと機会

• NPCにおけるアンメットニーズ
• 毒性が低いまたは最小の治療薬
• 投与のしやすさ
• NPC治療に対するコストの削減
• 有効性が向上した治療法
• ミグルスタットの承認

第6章 パイプラインの評価（2024年6月更新）

• NPCパイプラインの概要
• 主なパイプライン薬剤
• 製品プロファイル：アセチルロイシン、アドラベタデックス、トラップソルシクロ
• パイプライン製品 - レビュー指定
• NPC：臨床試験（フェーズII/III）の概要

第7章 研究開発戦略（2024年6月更新）

• NPCにおける臨床試験デザインの動向
• NPCにおける取引決定の動向

第8章 市場の見通し（2024年6月更新）

• NPC市場の予測
• 市場の促進要因と障壁

第9章 付録

第10章 お問い合わせ

The prevalence of Niemann Pick Type C (NPC) is increasing at a steady rate, in line with population growth in the 3MM at an AGR of 0.29%.

Currently, the treatment regimen for NPC in the 3MM is entirely limited to the use of miglustat (Yargesa, Zavesca or generic).

There are several unmet needs in this space, including therapies with low or minimal toxicity and novel therapies with improved efficacy.

The NPC late-stage R&D pipeline is focused on developing medicines with different mechanisms of action.

Steady growth is expected throughout the forecast period as the NPC population steadily increases and novel therapies enter the market.

Key Highlights

• Report deliverables include a Pdf and Excel-based forecast model
• Forecast includes three countries: US, Germany and the UK
• Forecasts cover three time points: base year, 5-year, and 10-year
• The prevalence of Niemann Pick Type C (NPC) is increasing at a steady rate, in line with population growth in the 3MM at an AGR of 0.29%.
• Currently, the treatment regimen for NPC in the 3MM is entirely limited to the use of miglustat (Yargesa, Zavesca or generic).
• There are several unmet needs in this space, including therapies with low or minimal toxicity and novel therapies with improved efficacy.
• The NPC late-stage R&D pipeline is focused on developing medicines with different mechanisms of action.
• Steady growth is expected throughout the forecast period as the NPC population steadily increases and novel therapies enter the market.

Scope

• Overview of NPC, including epidemiology, symptoms, diagnosis, and disease management.
• Annualized NPC therapeutics market revenue in 3MM (US, Germany and the UK), annual cost of therapy and treatment usage pattern in 2021 and forecast for 10 years to 2031; forecast covers three time points: base year, 5-year, and 10-year.
• Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the NPC therapeutics market.
• Pipeline analysis, comprehensive data assessing emerging trends and mechanisms of action under development for NPC treatment. The most promising candidates in late-stage development are profiled.
• Analysis of the current and future market competition in the 3MM NPC therapeutics market. Insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide a qualitative analysis of its implications.

Reasons to Buy

• Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
• Develop business strategies by understanding the trends shaping and driving the NPC therapeutics market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the NPC therapeutics market in the future.
• Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
• Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
• Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Table of Contents

Table of Contents

1 Executive Summary

• 1.1 Executive Summary

2 Disease Overview

• 2.1 Overview of Niemann-Pick Type C (NPC)
• 2.2 NPC SWOT Analysis
• 2.3 Classification of NPC

3 Epidemiology

• 3.1 Diagnosed Prevalent Cases of Niemann-Pick Type C, Men and Women, 2021-31
• 3.2 Sources and Methodology

4 Current Treatment Options

• 4.1 Treatment Paradigm
• 4.2 Marketed Products
• 4.3 Product Profiles

5 Unmet Needs and Opportunities

• 5.1 Unmet Needs in NPC
• 5.2 Therapies With Low or Minimal Toxicities
• 5.3 Ease of Administration
• 5.4 Reduced Cost for NPC Treatments
• 5.5 Therapies With Improved Efficacy
• 5.6 Miglustat Approval

6 Pipeline Assessment -Updated June 2024

• 6.1 NPC Pipeline Overview
• 6.2 Leading Pipeline Agents
• 6.3 Product Profiles: Acetylleucine, Adrabetadex, Trappsol Cyclo
• 6.4 Pipeline Products - Review Designations
• 6.5 NPC: Clinical Trials (Phase II/III) Overview

7 R&D Strategies - Updated June 2024

• 7.1 Trends in Clinical Trial Design in NPC
• 7.2 Trends in Deal-Making in NPC

8 Market Outlook - Updated June 2024

• 8.1 NPC Market Forecast
• 8.3 Market Drivers and Barriers

9 Appendix

• 9.1 Primary Research: KOL Information
• 9.2 About the Authors

10 Contact Us