查看购物车
  • Traditional Chinese
  • English
  • Japanese
封面
市场调查报告书
商品编码
2017491

斯特格氏黄斑失养症：市场展望、流行病学、竞争格局、市场预测报告（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个工作天内

价格
简介目录

市场概览

  • 目前，斯特格氏黄斑失养症市场规模小规模，主要集中在支持性治疗领域。预计到2025年，市场规模将达到4,670万美元，现有细分市场略有萎缩，但由于新产品的上市，整体市场预计将有所成长。
  • 在初期阶段，支持性治疗将占据主导地位，每年占 4,600 万至 4,700 万美元（2025-2028 年），但随着新疗法获得市场份额，预计到 2035 年，这一数字将下降到 2,500 万美元。
  • 从2030年起，在研疗法预计将产生可观的整体收入，具体而言：
  • AAVB-039 正成为主要的收入来源（到 2035 年约为 15 亿美元）。
  • 预计到 2035 年，OCU410ST 的销售额将达到 3.2 亿美元。
  • ALK-001 和 Emixustat 预计也将获得稳定的市场认可。
  • 整体市场正从低价值、分散的市场转变为高成长、创新主导的市场，其中基因疗法和细胞疗法正在推动价值呈指数级增长。

斯特格氏黄斑失养症概述

斯特格氏黄斑失养症(STGD1) 是最常见的遗传性青少年黄斑部病变病变，主要由 ABCA4 基因的体染色体隐性突变所引起。这种缺陷抑制了维生素 A 衍生物的转运，导致类视色素化合物（脂褐素）在视网膜色素上皮 (RPE) 中毒性累积。

由此导致的代谢功能障碍会引起进行性性黄斑萎缩和继发性感光细胞变性，临床表现为中心视力丧失、色觉障碍和暗适应障碍，通常始于儿童或青春期。

光学同调断层扫描(OCT) 和眼底萤光(FAF) 等先进影像技术用于诊断，可显示特征性视网膜斑块和黄斑部变薄。

目前尚无核准的疾病修正治疗，治疗主要以症状治疗为主。然而，治疗前景正在迅速发展，视觉循环调节剂、基因疗法和细胞疗法等研究阶段的方法正在探索中，所有这些方法都旨在抑制毒性代谢物的积累并维持视网膜功能。

主要亮点

  • 儘管斯特格氏黄斑失养症的新患者数量相对较少，但从 2025 年的 582 例稳步增加到 2035 年的 622 例，这反映了诊断方法和基因筛检的逐步改善。
  • 据估计，到 2025 年受影响的人数为 27,426 人，预计到 2035 年将增加到 29,343 人，这凸显了该疾病的慢性进行性性。
  • 接受治疗的患者人数仍然很高（治疗率为 89%），预计到 2035 年将从 24,409 人增加到 26,116 人，这主要是由于获得支持性护理和新疗法的机会增加。
  • 女性（62%）比男性（38%）更为普遍，而且这一趋势在整个预测期内保持不变。
  • 虽然该疾病的负担影响所有年龄层的人群，但中老年人（40-70 岁）的盛行率更高，这反映了该疾病随时间推移的累积进展。
  • 儘管目前已有治疗方法，但现有疗法无法解决潜在的遗传原因，也无法阻止疾病进展，因此仍存在巨大的未满足需求。

格式化和更新讯息

  • 详细报告（PDF）
  • 市场预测模型（基于微软Excel）
  • 流行病学数据（MS Excel，互动式工具）
  • 高阶主管洞察（PPT简报）
  • 其他功能：定期更新、自订和顾问支援。
  • 根据 Thelansis 的政策，我们确保所有最新更新在发布前都反映在报告内容和市场模型中。

主要问题

  • 我们如何优化 G8 市场（美国、欧盟 5 国、日本、中国）的药物开发与生命週期管理策略？
  • 从发病率、盛行率、人群组成以及接受药物治疗的患者人数来看，患者数量分别是多少？
  • 未来十年市场收入和病患份额的预测是多少？
  • 哪些因素对市场趋势影响最大？
  • 受访专家对目前和新兴疗法有何看法？
  • 哪款在研发线产品最有前景？其上市潜力及未来市场定位如何？
  • 主要未被满足的需求是什么？ KOL 对目标受众有何期望？
  • 为确保药物核准并顺利进入市场，必须满足哪些关键的监管和支付方要求？

目标国家

  • G8
    • 我们
    • EU5
      • 法国
      • 德国
      • 义大利
      • 西班牙
      • 英国
    • 日本
    • 中国

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.

目录

第一章：主要调查结果及分析师说明

  • 主要趋势：市场概况、SWOT分析、商业性利益与风险等。

第二章：疾病背景

  • 疾病定义、分类、病因和病理生理学、药物标靶等。

第三章：流行病学

  • 重点
  • 发病率/盛行率
  • 已确诊并接受药物治疗的患者人数
  • 合併症
  • 其他相关患者群

第四章 市场规模及预测

  • 重点
  • 市场驱动因素与限制因素
  • 按药物类别分類的趋势
  • 各国具体趋势

第五章 竞争情势

  • 目前疗法
    • 重点
    • 诊断和治疗过程/演算法
    • 主要疗法概述及KOL洞察
  • 新兴疗法
    • 重点
    • 值得关注的后期新疗法—概述、市场上市预期和KOL洞察
    • 值得关注的早期管道

第六章：未满足的需求与TPP分析

  • 主要未满足的需求以及透过新兴疗法实现的未来可能性
  • TPP分析与KOL展望

第七章 监理与报销环境

第八章附录

简介目录

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)