세계의 부신백질이영양증 치료제 : 시장 점유율과 순위, 전체 판매량 및 수요 예측(2025-2031년)

The global market for Adrenoleukodystrophy Drugs was estimated to be worth US$ 253 million in 2024 and is forecast to a readjusted size of US$ 591 million by 2031 with a CAGR of 10.9% during the forecast period 2025-2031.

Adrenoleukodystrophy (ALD) drugs are therapeutic solutions designed to prevent, slow, or reverse the neurodegenerative and endocrine manifestations caused by mutations in the ABCD1 gene, which lead to accumulation of very-long-chain fatty acids (VLCFAs) in the central nervous system and adrenal glands. The therapeutic landscape includes metabolic interventions (aimed at modulating fatty-acid metabolism and symptomatic support), hematopoietic stem cell transplant (HSCT)-related approaches that can arrest cerebral disease progression when performed early, and the more recent single-administration gene-replacement therapies that correct the underlying genetic defect in autologous cells. Clinical strategies differ markedly by phenotype - childhood cerebral ALD (CALD), adrenomyeloneuropathy (AMN), or adrenal insufficiency - and thus vary in mechanism of action, delivery modality, and regulatory pathway. Gene therapy introduces a potentially transformative one-time option for boys with early, active CALD who lack matched donors, while also imposing long-term safety monitoring and complex commercialization and reimbursement considerations. Overall, ALD drugs now span from conventional supportive regimens to advanced cell and gene therapeutics, reshaping clinical practice, payer evaluation, and R&D priorities in this ultra-rare disease area.

The ALD therapeutic space is experiencing an inflection point driven by the maturation of precision medicine and regulatory prioritization of rare-disease innovation. Regulatory acceleration and conditional approvals for one-time gene-replacement therapies have created a commercially viable pathway for treating boys with early, active CALD who lack matched donors-transforming clinical algorithms and motivating payers and providers to negotiate long-term outcome-based arrangements. Public health initiatives and expanded newborn or targeted screening programs are improving early detection rates, which in turn expand the addressable population for curative and high-value interventions. Companies that integrate R&D, manufacturing and Centers of Excellence into a coordinated delivery model can secure differentiated market access and durable clinical partnerships.

Innovation risk and commercialization complexity are prominent restraints. While gene therapies demonstrate compelling efficacy, long-term safety concerns-particularly vector integration-related hematologic malignancies-require extended monitoring and complicate regulatory labeling and payer coverage, thereby affecting adoption speed. Cross-border launches face heterogeneous reimbursement frameworks for ultra-rare, high-cost therapies; manufacturing and logistics for autologous cell-based products demand significant capital and operational capabilities. The patient population is inherently small and clinically heterogeneous, constraining rapid scale-up and return on investment. Company filings and regulatory communiques highlight that sponsors must manage clinical, regulatory and health-economic pressures in parallel.

Downstream demand is shifting toward an integrated "screen-treat-manage" ecosystem. Rising newborn and targeted screening rates will increase demand for early definitive interventions (HSCT or gene therapy), while families and clinicians will require robust long-term safety, quality-of-life, and functional-outcome data-driving growth in follow-up services and supportive care. Payers are increasingly receptive to innovative reimbursement constructs tied to long-term outcomes, prompting sponsors to design post-marketing evidence-generation strategies and scalable follow-up infrastructures. Consequently, procurement will evolve from single-product purchases toward bundled offerings that combine diagnostics, therapeutic delivery, and longitudinal outcomes management.

This report aims to provide a comprehensive presentation of the global market for Adrenoleukodystrophy Drugs, focusing on the total sales revenue, key companies market share and ranking, together with an analysis of Adrenoleukodystrophy Drugs by region & country, by Type, and by Application.

The Adrenoleukodystrophy Drugs market size, estimations, and forecasts are provided in terms of sales revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. With both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Adrenoleukodystrophy Drugs.

Market Segmentation

By Company

• Bluebird Bio
• NeuroVia
• Orpheris
• Minoryx
• MedDay Pharmaceuticals
• Rocket Pharmaceuticals
• Lysogene
• Sangamo Therapeutics

Segment by Type

• Hormone Replacement
• Transplant
• Others

Segment by Application

• Childhood Cerebral ALD
• Adrenomyeloneuropathy (AMN)
• Addison-only
• Others

By Region

• North America
  • United States
  • Canada
• Asia-Pacific
  • China
  • Japan
  • South Korea
  • Southeast Asia
  • India
  • Australia
  • Rest of Asia-Pacific
• Europe
  • Germany
  • France
  • U.K.
  • Italy
  • Netherlands
  • Nordic Countries
  • Rest of Europe
• Latin America
  • Mexico
  • Brazil
  • Rest of Latin America
• Middle East & Africa
  • Turkey
  • Saudi Arabia
  • UAE
  • Rest of MEA

Chapter Outline

Chapter 1: Introduces the report scope of the report, global total market size. This chapter also provides the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by manufacturers in the industry, and the analysis of relevant policies in the industry.

Chapter 2: Detailed analysis of Adrenoleukodystrophy Drugs company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.

Chapter 3: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.

Chapter 4: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.

Chapter 5: Revenue of Adrenoleukodystrophy Drugs in regional level. It provides a quantitative analysis of the market size and development potential of each region and introduces the market development, future development prospects, market space, and market size of each country in the world.

Chapter 6: Revenue of Adrenoleukodystrophy Drugs in country level. It provides sigmate data by Type, and by Application for each country/region.

Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product revenue, gross margin, product introduction, recent development, etc.

Chapter 8: Analysis of industrial chain, including the upstream and downstream of the industry.

Chapter 9: Conclusion.

Table of Contents

1 Market Overview

• 1.1 Adrenoleukodystrophy Drugs Product Introduction
• 1.2 Global Adrenoleukodystrophy Drugs Market Size Forecast (2020-2031)
• 1.3 Adrenoleukodystrophy Drugs Market Trends & Drivers
  • 1.3.1 Adrenoleukodystrophy Drugs Industry Trends
  • 1.3.2 Adrenoleukodystrophy Drugs Market Drivers & Opportunity
  • 1.3.3 Adrenoleukodystrophy Drugs Market Challenges
  • 1.3.4 Adrenoleukodystrophy Drugs Market Restraints
• 1.4 Assumptions and Limitations
• 1.5 Study Objectives
• 1.6 Years Considered

2 Competitive Analysis by Company

• 2.1 Global Adrenoleukodystrophy Drugs Players Revenue Ranking (2024)
• 2.2 Global Adrenoleukodystrophy Drugs Revenue by Company (2020-2025)
• 2.3 Key Companies Adrenoleukodystrophy Drugs Manufacturing Base Distribution and Headquarters
• 2.4 Key Companies Adrenoleukodystrophy Drugs Product Offered
• 2.5 Key Companies Time to Begin Mass Production of Adrenoleukodystrophy Drugs
• 2.6 Adrenoleukodystrophy Drugs Market Competitive Analysis
  • 2.6.1 Adrenoleukodystrophy Drugs Market Concentration Rate (2020-2025)
  • 2.6.2 Global 5 and 10 Largest Companies by Adrenoleukodystrophy Drugs Revenue in 2024
  • 2.6.3 Global Top Companies by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Adrenoleukodystrophy Drugs as of 2024)
• 2.7 Mergers & Acquisitions, Expansion

3 Segmentation by Type

• 3.1 Introduction by Type
  • 3.1.1 Hormone Replacement
  • 3.1.2 Transplant
  • 3.1.3 Others
• 3.2 Global Adrenoleukodystrophy Drugs Sales Value by Type
  • 3.2.1 Global Adrenoleukodystrophy Drugs Sales Value by Type (2020 VS 2024 VS 2031)
  • 3.2.2 Global Adrenoleukodystrophy Drugs Sales Value, by Type (2020-2031)
  • 3.2.3 Global Adrenoleukodystrophy Drugs Sales Value, by Type (%) (2020-2031)

4 Segmentation by Application

• 4.1 Introduction by Application
  • 4.1.1 Childhood Cerebral ALD
  • 4.1.2 Adrenomyeloneuropathy (AMN)
  • 4.1.3 Addison-only
  • 4.1.4 Others
• 4.2 Global Adrenoleukodystrophy Drugs Sales Value by Application
  • 4.2.1 Global Adrenoleukodystrophy Drugs Sales Value by Application (2020 VS 2024 VS 2031)
  • 4.2.2 Global Adrenoleukodystrophy Drugs Sales Value, by Application (2020-2031)
  • 4.2.3 Global Adrenoleukodystrophy Drugs Sales Value, by Application (%) (2020-2031)

5 Segmentation by Region

• 5.1 Global Adrenoleukodystrophy Drugs Sales Value by Region
  • 5.1.1 Global Adrenoleukodystrophy Drugs Sales Value by Region: 2020 VS 2024 VS 2031
  • 5.1.2 Global Adrenoleukodystrophy Drugs Sales Value by Region (2020-2025)
  • 5.1.3 Global Adrenoleukodystrophy Drugs Sales Value by Region (2026-2031)
  • 5.1.4 Global Adrenoleukodystrophy Drugs Sales Value by Region (%), (2020-2031)
• 5.2 North America
  • 5.2.1 North America Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 5.2.2 North America Adrenoleukodystrophy Drugs Sales Value by Country (%), 2024 VS 2031
• 5.3 Europe
  • 5.3.1 Europe Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 5.3.2 Europe Adrenoleukodystrophy Drugs Sales Value by Country (%), 2024 VS 2031
• 5.4 Asia Pacific
  • 5.4.1 Asia Pacific Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 5.4.2 Asia Pacific Adrenoleukodystrophy Drugs Sales Value by Region (%), 2024 VS 2031
• 5.5 South America
  • 5.5.1 South America Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 5.5.2 South America Adrenoleukodystrophy Drugs Sales Value by Country (%), 2024 VS 2031
• 5.6 Middle East & Africa
  • 5.6.1 Middle East & Africa Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 5.6.2 Middle East & Africa Adrenoleukodystrophy Drugs Sales Value by Country (%), 2024 VS 2031

6 Segmentation by Key Countries/Regions

• 6.1 Key Countries/Regions Adrenoleukodystrophy Drugs Sales Value Growth Trends, 2020 VS 2024 VS 2031
• 6.2 Key Countries/Regions Adrenoleukodystrophy Drugs Sales Value, 2020-2031
• 6.3 United States
  • 6.3.1 United States Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.3.2 United States Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.3.3 United States Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.4 Europe
  • 6.4.1 Europe Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.4.2 Europe Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.4.3 Europe Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.5 China
  • 6.5.1 China Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.5.2 China Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.5.3 China Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.6 Japan
  • 6.6.1 Japan Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.6.2 Japan Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.6.3 Japan Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.7 South Korea
  • 6.7.1 South Korea Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.7.2 South Korea Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.7.3 South Korea Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.8 Southeast Asia
  • 6.8.1 Southeast Asia Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.8.2 Southeast Asia Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.8.3 Southeast Asia Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031
• 6.9 India
  • 6.9.1 India Adrenoleukodystrophy Drugs Sales Value, 2020-2031
  • 6.9.2 India Adrenoleukodystrophy Drugs Sales Value by Type (%), 2024 VS 2031
  • 6.9.3 India Adrenoleukodystrophy Drugs Sales Value by Application, 2024 VS 2031

7 Company Profiles

• 7.1 Bluebird Bio
  • 7.1.1 Bluebird Bio Profile
  • 7.1.2 Bluebird Bio Main Business
  • 7.1.3 Bluebird Bio Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.1.4 Bluebird Bio Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.1.5 Bluebird Bio Recent Developments
• 7.2 NeuroVia
  • 7.2.1 NeuroVia Profile
  • 7.2.2 NeuroVia Main Business
  • 7.2.3 NeuroVia Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.2.4 NeuroVia Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.2.5 NeuroVia Recent Developments
• 7.3 Orpheris
  • 7.3.1 Orpheris Profile
  • 7.3.2 Orpheris Main Business
  • 7.3.3 Orpheris Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.3.4 Orpheris Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.3.5 Orpheris Recent Developments
• 7.4 Minoryx
  • 7.4.1 Minoryx Profile
  • 7.4.2 Minoryx Main Business
  • 7.4.3 Minoryx Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.4.4 Minoryx Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.4.5 Minoryx Recent Developments
• 7.5 MedDay Pharmaceuticals
  • 7.5.1 MedDay Pharmaceuticals Profile
  • 7.5.2 MedDay Pharmaceuticals Main Business
  • 7.5.3 MedDay Pharmaceuticals Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.5.4 MedDay Pharmaceuticals Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.5.5 MedDay Pharmaceuticals Recent Developments
• 7.6 Rocket Pharmaceuticals
  • 7.6.1 Rocket Pharmaceuticals Profile
  • 7.6.2 Rocket Pharmaceuticals Main Business
  • 7.6.3 Rocket Pharmaceuticals Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.6.4 Rocket Pharmaceuticals Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.6.5 Rocket Pharmaceuticals Recent Developments
• 7.7 Lysogene
  • 7.7.1 Lysogene Profile
  • 7.7.2 Lysogene Main Business
  • 7.7.3 Lysogene Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.7.4 Lysogene Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.7.5 Lysogene Recent Developments
• 7.8 Sangamo Therapeutics
  • 7.8.1 Sangamo Therapeutics Profile
  • 7.8.2 Sangamo Therapeutics Main Business
  • 7.8.3 Sangamo Therapeutics Adrenoleukodystrophy Drugs Products, Services and Solutions
  • 7.8.4 Sangamo Therapeutics Adrenoleukodystrophy Drugs Revenue (US$ Million) & (2020-2025)
  • 7.8.5 Sangamo Therapeutics Recent Developments

8 Industry Chain Analysis

• 8.1 Adrenoleukodystrophy Drugs Industrial Chain
• 8.2 Adrenoleukodystrophy Drugs Upstream Analysis
  • 8.2.1 Key Raw Materials
  • 8.2.2 Raw Materials Key Suppliers
  • 8.2.3 Manufacturing Cost Structure
• 8.3 Midstream Analysis
• 8.4 Downstream Analysis (Customers Analysis)
• 8.5 Sales Model and Sales Channels
  • 8.5.1 Adrenoleukodystrophy Drugs Sales Model
  • 8.5.2 Sales Channel
  • 8.5.3 Adrenoleukodystrophy Drugs Distributors

9 Research Findings and Conclusion

10 Appendix

• 10.1 Research Methodology
  • 10.1.1 Methodology/Research Approach
    • 10.1.1.1 Research Programs/Design
    • 10.1.1.2 Market Size Estimation
    • 10.1.1.3 Market Breakdown and Data Triangulation
  • 10.1.2 Data Source
    • 10.1.2.1 Secondary Sources
    • 10.1.2.2 Primary Sources
• 10.2 Author Details
• 10.3 Disclaimer