斯特格氏黃斑失養症：全球市場展望、流行病學、競爭格局和市場預測報告（32個主要市場），2025-2035年

市場概覽

• 目前，斯特格氏黃斑失養症市場規模小規模，主要集中在支持性治療領域。預計到2025年，市場規模將達到4,670萬美元，傳統治療領域將略有萎縮，但由於新藥開發平臺的推出，預計整體市場將有所成長。
• 在初期階段，支持性治療將佔主導地位，每年支出將達到 4,600 萬至 4,700 萬美元（2025-2028 年），但隨著新療法獲得市場佔有率，預計到 2035 年，這一數字將下降到 2,500 萬美元。
• 預計從 2030 年起，正在研發的療法將產生可觀的整體收入，具體如下：
• AAVB-039 正成為主要的收入來源（到 2035 年約為 15 億美元）。
• 預計到 2035 年，OCU410ST 的銷售額將達到 3.2 億美元。
• ALK-001 和 Emixustat 預計也將獲得穩定的市場滲透。
• 整體市場正從低價值、分散的市場向高成長、創新主導的市場轉變，其中基因療法和細胞療法推動了價值的急劇成長。

斯特格氏黃斑失養症概述

斯特格氏黃斑失養症是一種罕見的遺傳性黃斑營養不良，通常在兒童或青少年期發病，導致進行性中心視力喪失。最常見的原因是ABCA4基因突變。這種基因缺陷會破壞感光細胞內維生素A衍生物的正常轉運，導致有毒的脂褐素在視網膜色素上皮中累積。結果，負責清晰、精細視覺的視網膜部分——黃斑部——受到損害。患者通常會出現視力模糊、閱讀困難和畏光等症狀，而周邊視力通常得以保留。此疾病呈現體染色體隱性遺傳模式，可透過眼底影像檢查、光學同調斷層掃描（OCT）、視網膜電圖和基因檢測進行診斷。

主要亮點

• 儘管斯特格氏黃斑失養症的新病例數量相對較低，但從 2025 年的 582 例穩步增加到 2035 年的 622 例，這反映了診斷技術和基因篩檢的逐步改進。
• 據估計，到 2025 年受影響的人數為 27,426 人，預計到 2035 年將增加到 29,343 人，這凸顯了該疾病的慢性進行性性。
• 接受治療的患者人數仍然很高（治療率為 89%），預計將從 2025 年的 24,409 人增加到 2035 年的 26,116 人。這主要是由於獲得支持性護理和新療法的機會增加。
• 女性（62%）人數超過男性（38%），而且這一趨勢在整個預測期內保持不變。
• 此疾病的負擔遍及所有年齡層，但中老年人（40-70 歲）的盛行率較高，反映了疾病隨時間推移的累積進展。
• 儘管目前已有治療方法，但現有療法無法解決潛在的遺傳原因，也無法阻止疾病進展，因此仍存在巨大的未滿足需求。

市場定義：

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

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

• 我們如何最佳化在 32 個市場（北美、歐洲、中東、亞太、非洲和南美/拉丁美洲）的藥物開發和生命週期管理策略？
• 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？
• 為確保藥物獲得批准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• 北美洲（美國、加拿大）
• 歐洲（奧地利、比利時、捷克、丹麥、芬蘭、法國、德國、希臘、義大利、荷蘭、挪威、波蘭、葡萄牙、俄羅斯、西班牙、瑞典、瑞士、英國）
• 中東（沙烏地阿拉伯、阿拉伯聯合大公國、科威特、土耳其）
• 亞太地區（澳洲、中國、香港、印度、印尼、日本、馬來西亞、紐西蘭、菲律賓、新加坡、韓國、台灣、泰國、越南）
• 非洲（埃及、奈及利亞、南非、摩洛哥）
• 南美洲/拉丁美洲（阿根廷、巴西、智利、哥倫比亞、墨西哥、秘魯）

大公司

• Sanofi
• Alkeus Pharmaceuticals, Inc.
• AAVantgarde Bio Srl
• Ray Therapeutics, Inc.
• Ocugen
• Splice Bio
• Heronova Pharmaceuticals
• Belite Bio, Inc
• Ascidian Therapeutics, Inc
• VeonGen Therapeutics GmbH
• Astellas Pharma Global Development, Inc.

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
• 已確診並正在接受藥物治療的患者族群。
• 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
• 目前主要療法－概述及關鍵意見領袖見解
• 新興療法
• 重點
• 值得關注的後期新興療法—概述、市場上市預期和關鍵意見領袖見解
• 值得關注的早期管道

第6章：未滿足的需求與TPP分析

• 尚未滿足的主要需求及其透過新興療法實現的潛力。
• TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

Global Stargardt Disease Market Outlook

Thelansis's "Global Stargardt Disease Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report (Top 32 Markets) - 2025 To 2035" covers disease overview, epidemiology, kidney biopsy cases, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Stargardt Disease treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Stargardt Disease Overview

Stargardt disease is a rare, inherited macular dystrophy that leads to progressive central vision loss, typically beginning in childhood or adolescence, and is most commonly caused by mutations in the ABCA4 gene. This genetic defect disrupts the normal transport of vitamin A derivatives in photoreceptor cells, resulting in the accumulation of toxic lipofuscin within the retinal pigment epithelium, which damages the macula-the part of the retina responsible for sharp, detailed vision. Patients often report blurred vision, difficulty reading, and increased sensitivity to light, while peripheral vision usually remains preserved. The condition follows an autosomal recessive inheritance pattern and is diagnosed through fundus imaging, optical coherence tomography (OCT), electroretinography, and genetic testing.

Key Highlights

• The incident Stargardt disease population is relatively small but steadily increasing, from 582 cases in 2025 to 622 cases by 2035, reflecting gradual improvements in diagnosis and genetic screening.
• The prevalent population is estimated at 27,426 cases in 2025, increasing to 29,343 by 2035, highlighting the chronic and progressive nature of the disease.
• The treated patient population remains high (89% treatment rate), increasing from 24,409 patients in 2025 to 26,116 by 2035, largely driven by supportive care and emerging therapy access.
• A female predominance (62%) is observed compared to males (38%), consistent across the forecast period.
• Disease burden spans across age groups but shows higher prevalence in middle-aged and older cohorts (40-70 years), reflecting cumulative disease progression over time.
• Despite treatment availability, there remains a significant unmet need, as current therapies do not address the underlying genetic cause or halt disease progression.

Market Overview

• The Stargardt disease market is currently small and largely supportive-care driven, with total market size estimated at $46.7M in 2025, projected to decline slightly in legacy segments but expand overall with pipeline entry.
• Supportive therapies dominate early years, contributing $46-47M annually (2025-2028), but decline to $25M by 2035 as novel therapies gain share.
• Pipeline therapies collectively generate substantial revenue post-2030, with:
• AAVB-039 emerging as a dominant contributor (~$1.5Bn by 2035)
• OCU410ST reaching $320M by 2035
• ALK-001 and Emixustat also showing steady uptake
• The total market transitions from a low-value, fragmented landscape to a high-growth, innovation-driven market, with gene and cell therapies driving exponential value expansion.

Market Definition:

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

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 the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America)?
• 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

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Companies Mentioned

• Sanofi
• Alkeus Pharmaceuticals, Inc.
• AAVantgarde Bio Srl
• Ray Therapeutics, Inc.
• Ocugen
• Splice Bio
• Heronova Pharmaceuticals
• Belite Bio, Inc
• Ascidian Therapeutics, Inc
• VeonGen Therapeutics GmbH
• Astellas Pharma Global Development, Inc.

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)