全球腎上腺腦白質失養症市場：按地區和國家分析和預測（2025年至2035年）

全球腎上腺腦白質失養症市場目前正處於其生命週期的成長階段。

在經歷了治療方法有限和認知度較低的導入期之後，隨著基於基因療法的治療方法和創新藥物療法的進步，市場對該疾病的興趣日益濃厚。新生兒篩檢計劃等診斷技術的改進使得更早發現疾病成為可能，增加了對治療性介入的需求。此外，孤兒藥計畫和政府對罕見疾病的支持力度也在推動市場成長。然而，市場仍然面臨著若干挑戰，最明顯的是治療費用高昂以及由於患者數量相對較少導致的市場規模有限。這些因素可能會阻礙市場的快速擴張。總體而言，市場目前成長但仍高度專業化，預計持續的研究和開發將促進整個罕見疾病領域的發展。

影響

• 預計預測期內，對腎上腺腦白質失養症治療的需求不斷成長將推動全球腎上腺腦白質失養症市場的成長
• 由於標靶治療的進步、認知度和診斷率的提高、臨床開發平臺的擴大、醫療基礎設施的改善以及專科治療機會的改善，全球 ALD 市場預計將在未來顯著成長。

近期動態

• 研究活動：2024年 12月，Minoryx Therapeutics 和 Neuraxpharm Group 宣布，leriglitazone 在針對患有大腦腎上腺腦白質失養症的兒科患者的NEXUS 研究中達到了其主要終點。

本報告調查了全球腎上腺腦白質失養症市場，並總結了主要趨勢、影響市場的因素分析、法律制度、臨床試驗趨勢、市場規模趨勢和預測、各個細分市場、地區/主要國家的詳細分析、競爭格局和主要企業的概況。

目錄

執行摘要

第1章 全球腎上腺腦白質失養症市場概述

• 產業展望
  • 市場概況與生態系統
  • 市場趨勢
  • 腎上腺腦白質失養症的流行病學分析
  • 臨床試驗
  • 監管狀況/合規性
• 市場動態
  • 影響分析
  • 市場促進因素
  • 市場限制
  • 市場機會

第2章 全球腎上腺腦白質失養症市場（依地區）

• 北美洲
• 歐洲
• 亞太地區
  • 市場動態
  • 市場規模及預測

第3章 全球腎上腺腦白質失養症市場：競爭基準化分析與公司簡介

• 競爭格局
  • 各公司主要策略及發展
  • 主要發展分析
• 公司簡介
  • bluebird bio
  • Minoryx Therapeutics, SL
  • MedDay Pharmaceuticals
  • Viking Therapeutics
  • Neuraxpharm Group

第4章 調查方法

Industry Overview

The global adrenoleukodystrophy market is currently in the growth stage of its lifecycle. Having transitioned from the introduction phase, where treatment options were limited and awareness was low, the market is now experiencing increased interest due to advances in gene therapy-based treatments and innovative drug therapies. Rising diagnostic capabilities, such as newborn screening programs, have contributed to earlier detection, driving demand for therapeutic interventions. Furthermore, the emergence of orphan drug programs and government support for rare diseases has spurred market growth. However, the market still faces challenges, including high treatment costs and a relatively small patient base, which may limit rapid expansion. Overall, while the market is growing, it remains highly specialized and will continue to evolve the rare disease landscape with ongoing research and development.

Impact

• Increasing demand for adrenoleukodystrophy therapies is anticipated to support the growth of the global adrenoleukodystrophy market during the forecast period 2025-2035.
• The global adrenoleukodystrophy market is expected to grow at a significant rate due to developments in targeted therapies, rising awareness and diagnosis, and a growing clinical pipeline, alongside advancements in healthcare infrastructure and access to specialized treatments.

Recent Developments

• Research Activities: In December 2024, Minoryx Therapeutics and Neuraxpharm Group announced that leriglitazone met the primary endpoint in the NEXUS trial for paediatric patients with cerebral adrenoleukodystrophy (cALD).

Demand - Drivers and Limitations

Drivers:

• Increasing Prevalence of Adrenoleukodystrophy
• Advancements in Treatment Options
• Growing Research and Investment in Rare Diseases

Limitations:

• High Treatment Costs
• Limited Awareness in Developing Regions

How Can This Report Add Value to an Organization?

Product/Innovation Strategy: Innovations in the global adrenoleukodystrophy market are centered on advancing treatment options to enhance patient care. Key players in the market, such as Minoryx Therapeutics and NeuraxPharm, have been involved in the development of therapies for adrenoleukodystrophy.

Competitive Strategy: Enterprises led by market leaders continually update their product portfolios with innovative treatments to maintain competitiveness. A detailed competitive benchmarking of the key players has been conducted, providing insights into how these companies compare in terms of product pipeline and innovation. This benchmarking provides readers with a clear understanding of the market landscape and the positions of the leading players. Additionally, comprehensive competitive strategies, such as partnerships, agreements, and collaborations, will help readers identify untapped revenue opportunities in the market.

Key Market Players and Competition Synopsis

The companies profiled have been selected based on inputs gathered from primary experts and by analyzing company coverage, product portfolios, and market penetration.

Some of the prominent names established in this market are:

• bluebird bio
• Minoryx Therapeutics, S.L.
• MedDay Pharmaceuticals
• Viking Therapeutics
• Neuraxpharm Group

Table of Contents

Executive Summary

Scope of Study

1. Global Adrenoleukodystrophy Market Overview

• 1.1 Industry Outlook
  • 1.1.1 Market Overview and Ecosystem
  • 1.1.2 Market Trends
  • 1.1.3 Epidemiological Analysis of Adrenoleukodystrophy
  • 1.1.4 Clinical Trials
    • 1.1.4.1 By Phase
    • 1.1.4.2 By Sponsor Type
  • 1.1.5 Regulatory Landscape / Compliance
• 1.2 Market Dynamics
  • 1.2.1 Impact Analysis
  • 1.2.2 Market Drivers
  • 1.2.3 Market Restraints
  • 1.2.4 Market Opportunities

2. Global Adrenoleukodystrophy Market (By Region), $Million, 2023-2035

• 2.1 North America
  • 2.1.1 Market Dynamics
  • 2.1.2 Market Size and Forecast
    • 2.1.2.1 North America Adrenoleukodystrophy Market (by Country)
      • 2.1.2.1.1 U.S.
• 2.2 Europe
  • 2.2.1 Market Dynamics
  • 2.2.2 Market Size and Forecast
    • 2.2.2.1 Europe Adrenoleukodystrophy Market (by Country)
      • 2.2.2.1.1 U.K.
      • 2.2.2.1.2 Germany
      • 2.2.2.1.3 France
      • 2.2.2.1.4 Italy
      • 2.2.2.1.5 Spain
• 2.3 Asia-Pacific
  • 2.3.1 Market Dynamics
  • 2.3.2 Market Size and Forecast
    • 2.3.2.1 Asia-Pacific Adrenoleukodystrophy Market (by Country)
      • 2.3.2.1.1 Japan

3. Global Adrenoleukodystrophy Market - Competitive Benchmarking and Company Profiles

• 3.1 Competitive Landscape
  • 3.1.1 Key Strategies and Developments by Company
    • 3.1.1.1 Funding Activities
    • 3.1.1.2 Mergers and Acquisitions
    • 3.1.1.3 Regulatory Approvals
    • 3.1.1.4 Partnerships, Collaborations and Business Expansions
  • 3.1.2 Key Developments Analysis
• 3.2 Company Profiles
  • 3.2.1 bluebird bio
    • 3.2.1.1 Company Overview
    • 3.2.1.2 Product Portfolio
    • 3.2.1.3 Target Customers/End Users
    • 3.2.1.4 Analyst View
  • 3.2.2 Minoryx Therapeutics, S.L.
    • 3.2.2.1 Company Overview
    • 3.2.2.2 Product Portfolio
    • 3.2.2.3 Target Customers/End Users
    • 3.2.2.4 Analyst View
  • 3.2.3 MedDay Pharmaceuticals
    • 3.2.3.1 Company Overview
    • 3.2.3.2 Product Portfolio
    • 3.2.3.3 Target Customers/End Users
    • 3.2.3.4 Analyst View
  • 3.2.4 Viking Therapeutics
    • 3.2.4.1 Company Overview
    • 3.2.4.2 Product Portfolio
    • 3.2.4.3 Target Customers/End Users
    • 3.2.4.4 Analyst View
  • 3.2.5 Neuraxpharm Group
    • 3.2.5.1 Company Overview
    • 3.2.5.2 Product Portfolio
    • 3.2.5.3 Target Customers/End Users
    • 3.2.5.4 Analyst View

4. Research Methodology

List of Figures

• Figure: Global Adrenoleukodystrophy Market, Market Overview
• Figure: Global Adrenoleukodystrophy Market, Epidemiological Analysis, U.S.
• Figure: Global Adrenoleukodystrophy Market Coverage
• Figure: Global Adrenoleukodystrophy Market Key Trends, Impact Analysis, 2023-2035
• Figure: Global Adrenoleukodystrophy Market, Competitive Landscape, January 2022-April 2025

List of Tables

• Table: Global Adrenoleukodystrophy Market, Regulatory Scenario
• Table: Global Adrenoleukodystrophy Market Dynamics, Impact Analysis
• Table: Global Adrenoleukodystrophy Market (by Region), $Million, 2023-2035