亨廷頓舞蹈症：市場展望、流行病學、競爭格局、市場預測報告（2025-2035年）

市場概覽

• 德國亨廷頓舞蹈症市場預計將從約 4,200 萬美元成長到 1.3 億美元，這表明市場將大幅擴張。
• 市場成長受以下因素支撐：
• 擴大症狀治療和多學科疾病管理
• 基因靜默與疾病修正治療的發展進展
• 人們對診斷的認知不斷提高，以及基因檢測的普及，都促進了患者群體的擴大。
• 未來市場成長將很大程度上取決於針對突變亨廷頓蛋白路徑的下一代疾病修正治療的商業化成功。

亨丁頓舞蹈症概述

亨廷頓氏舞蹈症（HD）是一種罕見的、進行性的體染色體顯性遺傳神經退化性疾病，由HTT基因中CAG三核苷酸重複序列的擴增引起，導致產生有毒的突變亨廷頓蛋白（mHTT）。此病以神經元進行性性退化為特徵，尤其是在紋狀體和大腦皮層，最終導致運動障礙、認知功能下降和精神障礙加重。此病常有遺傳領先，每一代都有發病年齡提前和病情加重的趨勢。

臨床上，亨丁頓舞蹈症主要表現為三種症狀：運動障礙、認知障礙、精神障礙。早期通常表現為舞蹈症等運動過度，但隨著晚期，會發展為肌張力增高、肌張力不全症、協調障礙和嚴重的機能衰退。認知障礙逐漸進展為皮質下失智症，並伴隨憂鬱症、冷漠、焦慮、易怒和衝動行為等精神症狀，這些症狀嚴重影響患者的生活品質，加重看護者的負擔。

目前，尚無核准的根治性或疾病修飾療法治療方法阻止或逆轉疾病進展，治療仍以症狀支持為主。 VMAT2抑制劑，包括Tetrabenazine和Tetrabenazine，常用於治療舞蹈症，而抗憂鬱症、抗精神病藥物和多學科支持治療則用於治療精神症狀。隨著基因靜默療法、RNA標靶療法和緩解疾病策略日益受到關注，這些療法有望徹底改變亨丁頓舞蹈症未來的治療模式。

主要亮點

• 在美國，亨廷頓舞蹈症的診斷數量預計將從約 39,486 例增加到 42,458 例，反映出確診患者數量的逐漸增加。
• HD仍然是神經退化性疾病，由於沒有核准的疾病修正治療，因此存在很大的未滿足需求。
• 運動障礙、認知能力下降和精神併發症持續顯著加重疾病負擔和對照護的依賴。
• 隨著新的基因標靶治療和基於 RNA 的治療方法的出現，人們對血液透析 (HD) 治療的研究和開發的興趣顯著成長。
• 人們意識的提高和基因檢測技術的進步，使得患者的診斷和識別能夠更早進行。

格式化和更新訊息

• 詳細報告（PDF）
• 市場預測模型（基於微軟Excel）
• 流行病學數據（MS Excel，互動式工具）
• 高階主管洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，我們確保所有最新更新在發布前都反映在報告內容和市場模型中。

主要問題

• 我們如何最佳化 G8 市場（美國、歐盟 5 國、日本、中國）的藥物開發與生命週期管理策略？
• 從發病率、盛行率、人群組成以及接受藥物治療的患者人數來看，患者數量分別是多少？
• 未來十年市場收入和病患佔有率的預測是多少？
• 哪些因素對市場趨勢影響最大？
• 受訪專家對目前和新興的治療方法有何看法？
• 哪款在研發線產品最有前景？其上市潛力及未來市場定位如何？
• 主要未被滿足的需求是什麼？ KOL 對目標受眾有何期望？
• 為確保藥物核准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• G8
  • 美國
  • EU5
    • 法國
    • 德國
    • 義大利
    • 西班牙
    • 英國
  • 日本
  • 中國

大公司

• SOM Innovation Biotech SA
• Neurocrine Biosciences
• Skyhawk Therapeutics, Inc.
• Sarepta Therapeutics, Inc.
• Hoffmann-La Roche
• Alnylam Pharmaceuticals
• Supernus Pharmaceuticals, Inc.
• UniQure Biopharma BV
• Novartis Pharmaceuticals
• Prilenia
• ExoRNA Bioscience
• Medibiofarma SL
• Wave Life Sciences Ltd.
• PTC Therapeutics

目錄

第1章：主要調查結果及分析師說明

• 主要趨勢：市場概況、SWOT分析、商業性利益與風險等。

第2章：疾病背景

• 疾病定義、分類、病因和病理生理學、藥物標靶等。

第3章：流行病學

• 重點
• 發病率/盛行率
• 已確診並接受藥物治療的患者人數
• 合併症
• 其他相關患者群

第4章 市場規模及預測

• 重點
• 市場促進因素與限制因素
• 按藥物類別分類的趨勢
• 各國具體趨勢

第5章 競爭情勢

• 目前的治療方法
• 重點
• 診斷和治療過程/演算法
• 主要治療方法概述及KOL洞察
• 新興治療方法
• 重點
• 值得關注的後期新治療方法－概述、市場上市預期及KOL洞察
• 值得關注的早期研發管線

第6章：未滿足的需求與TPP分析

• 主要未滿足的需求及其未來透過新興治療方法實現的可能性
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Huntington Disease Market Outlook

Thelansis's "Huntington Disease Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Huntington Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Huntington's Disease Overview

Huntington's disease (HD) is a rare, progressive, autosomal dominant neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the HTT gene, resulting in the production of toxic mutant huntingtin (mHTT) protein. The disease is characterized by progressive degeneration of neurons, particularly within the striatum and cerebral cortex, leading to worsening motor dysfunction, cognitive decline, and psychiatric disturbances. Genetic anticipation is commonly observed, with successive generations often experiencing earlier disease onset and increased severity.

Clinically, HD presents with a triad of motor, cognitive, and psychiatric symptoms. Early disease is typically associated with hyperkinetic movements such as chorea, while advanced stages progress toward rigidity, dystonia, impaired coordination, and severe functional decline. Cognitive impairment gradually evolves into subcortical dementia, accompanied by psychiatric manifestations including depression, apathy, anxiety, irritability, and impulsive behavior, significantly impacting patient quality of life and caregiver burden.

Currently, no curative or disease-modifying therapies are approved to halt or reverse disease progression, and management remains primarily symptomatic and supportive. VMAT2 inhibitors, including tetrabenazine and deutetrabenazine, are commonly used to manage chorea, while psychiatric symptoms are addressed through antidepressants, antipsychotics, and multidisciplinary supportive care. Increasing research focus on gene-silencing therapies, RNA-targeted approaches, and disease-modifying strategies is expected to reshape the future treatment landscape for HD.

Key Highlights

• In the USA, diagnosed Huntington's disease cases are projected to increase from approximately 39,486 to 42,458, reflecting a gradually increasing diagnosed patient population.
• HD remains a high unmet need neurodegenerative disorder due to the absence of approved disease-modifying therapies.
• Motor dysfunction, cognitive decline, and psychiatric complications continue to contribute substantially to disease burden and caregiver dependency.
• Emerging gene-targeting and RNA-based therapies are driving significant research and clinical development interest in HD treatment.
• Increasing awareness and improved genetic testing are supporting earlier diagnosis and patient identification.

Market Overview

• The Germany Huntington's disease market is projected to grow from approximately $42 MN to $130 MN, reflecting significant market expansion.
• Market growth is supported by:
• Increasing adoption of symptomatic therapies and multidisciplinary disease management
• Advancing development of gene-silencing and disease-modifying therapeutic approaches
• Rising diagnostic awareness and improved access to genetic testing are contributing to patient pool expansion.
• Future market growth will largely depend on successful commercialization of next-generation disease-modifying therapies targeting mutant huntingtin protein pathways.

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• SOM Innovation Biotech SA
• Neurocrine Biosciences
• Skyhawk Therapeutics, Inc.
• Sarepta Therapeutics, Inc.
• Hoffmann-La Roche
• Alnylam Pharmaceuticals
• Supernus Pharmaceuticals, Inc.
• UniQure Biopharma B.V.
• Novartis Pharmaceuticals
• Prilenia
• ExoRNA Bioscience
• Medibiofarma S.L.
• Wave Life Sciences Ltd.
• PTC Therapeutics

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)