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市場調查報告書
商品編碼
2017491

斯特格氏黃斑失養症：市場展望、流行病學、競爭格局、市場預測報告（2025-2035年）

Stargardt Disease - Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035
出版日期: | 出版商: Thelansis Knowledge Partners | 英文 154 Pages | 商品交期: 2-3個工作天內

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簡介目錄

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，現有細分市場略有萎縮，但由於新產品的上市，整體市場預計將有所成長。
  • 在初期階段，支持性治療將佔據主導地位，每年佔 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。
  • 從2030年起，在研療法預計將產生可觀的整體收入，具體而言：
  • 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認可。
  • 整體市場正從低價值、分散的市場轉變為高成長、創新主導的市場，其中基因療法和細胞療法正在推動價值呈指數級成長。

斯特格氏黃斑失養症概述

斯特格氏黃斑失養症(STGD1) 是最常見的遺傳性青少年黃斑部病變病變，主要由 ABCA4 基因的體染色體隱性突變所引起。這種缺陷抑制了維生素 A 衍生物的轉運，導致類視色素化合物（脂褐素）在視網膜色素上皮 (RPE) 中毒性累積。

由此導致的代謝功能障礙會引起進行性性黃斑萎縮和繼發性感光細胞變性，臨床表現為中心視力喪失、色覺障礙和暗適應障礙，通常始於兒童或青春期。

光學同調斷層掃描(OCT) 和眼底螢光(FAF) 等先進影像技術用於診斷，可顯示特徵性視網膜斑塊和黃斑部變薄。

目前尚無核准的疾病修正治療，治療主要以症狀治療為主。然而，治療前景正在迅速發展，視覺循環調節劑、基因療法和細胞療法等研究階段的方法正在探索中，所有這些方法都旨在抑制毒性代謝物的積累並維持視網膜功能。

主要亮點

  • 儘管斯特格氏黃斑失養症的新患者數量相對較少，但從 2025 年的 582 例穩步增加到 2035 年的 622 例，這反映了診斷方法和基因篩檢的逐步改善。
  • 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%），預計到 2035 年將從 24,409 人增加到 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%）比男性（38%）更為普遍，而且這一趨勢在整個預測期內保持不變。
  • 雖然該疾病的負擔影響所有年齡層的人群，但中老年人（40-70 歲）的盛行率更高，這反映了該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。

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療法有何看法？
  • 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

  • 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

  • 目前療法
    • 重點
    • 診斷和治療過程/演算法
    • 主要療法概述及KOL洞察
  • 新興療法
    • 重點
    • 值得關注的後期新療法—概述、市場上市預期和KOL洞察
    • 值得關注的早期管道

第6章：未滿足的需求與TPP分析

  • 主要未滿足的需求以及透過新興療法實現的未來可能性
  • TPP分析與KOL展望

第7章 監理與報銷環境

第8章附錄

簡介目錄

Stargardt Disease Market Outlook

Thelansis's "Stargardt 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 Stargardt Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Stargardt Disease Overview

Stargardt disease (STGD1) is the most common form of inherited juvenile macular degeneration, primarily caused by autosomal recessive mutations in the ABCA4 gene. This defect disrupts the transport of vitamin A derivatives, leading to toxic accumulation of bisretinoid compounds (lipofuscin) within the retinal pigment epithelium (RPE).

The resulting metabolic dysfunction drives progressive macular atrophy and secondary photoreceptor degeneration, clinically manifesting as loss of central vision, impaired color perception, and difficulty with dark adaptation, typically presenting in childhood or adolescence.

Diagnosis is supported by advanced imaging modalities such as optical coherence tomography (OCT) and fundus autofluorescence (FAF), which reveal characteristic retinal flecks and macular thinning.

Currently, there are no approved disease-modifying therapies, and management is largely supportive. However, the therapeutic landscape is evolving rapidly, with investigational approaches including visual cycle modulators, gene therapies, and cell-based treatments aimed at reducing toxic metabolite accumulation and preserving retinal function.

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.

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/report will cost USD 1,750 per country

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)