副腎白質ジストロフィー治療の世界市場：2024年～2031年

概要

世界の副腎白質ジストロフィー治療市場は、2023年に3億2,336万米ドルに達し、2031年には6億7,303万米ドルに達すると予測され、予測期間2024年にはCAGR 9.6%で成長する見込みです。

アドレナリン白質ジストロフィーは、X染色体上にあるABCD1遺伝子の変異によって引き起こされる疾患です。神経変性疾患であり、脳と脊髄の神経線維を覆う保護ミエリン鞘が徐々に劣化します。この脱髄により脳が傷害され、患者に見られる行動学的、神経学的症状を引き起こします。この病気は副腎にも影響を及ぼし、副腎で十分なステロイドホルモンが産生されなくなります。

副腎白質ジストロフィーの最も一般的なタイプは、X染色体上の遺伝子異常によって起こるX連鎖性ALDです。X連鎖性ALDは、X染色体上の遺伝子異常によって発症します。世界の副腎白質ジストロフィー治療市場は、幹細胞移植治療の採用により近年著しい成長を遂げています。同種造血幹細胞移植（HSCT）は、X連鎖性アドレノロイコジストロフィーの脳脱髄を阻止する唯一の治療アプローチです。

市場力学：

促進要因

アドレナリン白質ジストロフィー治療薬の研究開発とFDA承認の増加

世界の副腎白質ジストロフィー治療市場の需要は、複数の要因によって牽引されています。主な要因の1つは、研究開発の増加と治療薬のFDA承認の増加です。

副腎白質ジストロフィー（ALD）は、神経系の白質と副腎皮質を侵すまれな遺伝性疾患です。2022年9月のNational Organization for Rare Disordersによると、ALDの有病率は一般人口で10,000人から17,000人に1人と推定されています。

さらに、政府の取り組みや、副腎白質ジストロフィーの治療における先進的な研究開発が、この市場の成長を後押ししています。例えば、2023年8月、Orpheris社は、薬剤アセチルシステインジドリマーがX連鎖性アドレノロイコジストロフィー（X-ALD）に対して第II相臨床開発中であることを発表しました。

さらに、提携・協力や製品上市・承認などの業界戦略における主要企業が、この市場成長を促進すると思われます。たとえば、2022年11月、Minoryx TherapeuticsとNeuraxpharmは、X-ALD成人男性患者の治療におけるレリグリタゾンの承認を取得するための欧州規制プロセスの締結に協力しました。レリグリタゾンは、欧州医薬品庁 (EMA) によって承認された最初の治療法です。

2022年5月、POXEL SAは、米国食品医薬品局（FDA）が、X連鎖性副腎白質ジストロフィー（ALD）の最も一般的な病型である副腎神経根症（AMN）患者の治療薬として、PXL065とPXL770に希少疾病用医薬品指定（ODD）を付与したと発表しました。

抑制要因

高額な治療費、幹細胞移植に伴う副作用、限られた治療選択肢、人々の認識不足などの要因が、市場の抑制要因になると予想されます。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 副腎白質ジストロフィー治療の研究開発とFDA承認の増加
    • X連鎖性劣性疾患の症例増加
  • 抑制要因
    • 治療費の高騰
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• アンメットニーズ
• 規制分析
• DMIの見解

第6章 タイプ別

• 小児期脳性ALD
• 副腎脊髄神経障害（AMN）
• アジソン病

第7章 治療法別

• 幹細胞移植
• 副腎皮質ステロイド補充療法
• 薬物療法
• 遺伝子治療
• ロレンツォオイル
• その他

第8章 エンドユーザー別

• 病院
• 専門クリニック
• その他

第9章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
    • 英国
    • フランス
    • イタリア
    • スペイン
    • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
    • インド
    • 日本
    • 韓国
    • その他アジア太平洋
• 中東・アフリカ

第10章 競合情勢

• 競合シナリオ
• 製品ベンチマーク
• 企業シェア分析
• 主な発展と戦略

第11章 企業プロファイル

• Bluebird Bio Inc.
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Viking Therapeutics
• Magenta Therapeutics
• NeuroVia
• Minoryx Therapeutics
• Poxel S.A.
• Orpheris
• SwanBio Therapeutics
• Sperogenix Therapeutics
• Autobahn Therapeutics

第12章 付録

Overview

The global adrenoleukodystrophy treatment market reached US$ 323.36 million in 2023 and is expected to reach US$ 673.03 million by 2031 growing with a CAGR of 9.6% during the forecast period 2024-2031.

Adrenoleukodystrophy is a disease caused by a mutation in the ABCD1 gene that is located on the X chromosome. It is a neurodegenerative disease in which the protective myelin sheath covering the nerve fibers in the brain and spinal cord gradually deteriorates. This demyelination causes injury to the brain leading to the behavioral and neurological symptoms seen in the patients. The disease also affects the adrenal glands causing them not to produce enough steroid hormones.

The most common type of adrenoleukodystrophy is X-linked ALD caused by a genetic defect on the X chromosome. It mostly affects males more severely than females. The global adrenoleukodystrophy treatment market has witnessed significant growth in recent years due to the adoption of stem cell transplantation treatments. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only therapeutic approach to arrest cerebral demyelination of X-linked adrenoleukodystrophy.

Market Dynamics: Drivers

Increasing Research & Developments and Rising FDA Approvals of Drugs for Adrenoleukodystrophy Treatment

The demand for the global adrenoleukodystrophy treatment market is driven by multiple factors. One of the primary factors is the increasing research & developments and rising FDA approvals of drugs for the treatment.

Adrenoleukodystrophy (ALD) is a rare genetic disorder that affects the white matter of the nervous system and the adrenal cortex. According to the National Organization for Rare Disorders in September 2022, the prevalence of ALD is estimated to be between 1 in 10,000 and 1 in 17,000 individuals in the general population.

Moreover, government initiatives and advanced research and developments in treating adrenoleukodystrophy help to drive this market growth. For instance, in August 2023, Orpheris announced that the drug Acetylcysteine zidrimer is under clinical development in Phase II for X-linked adrenoleukodystrophy (X-ALD).

In addition, key players in the industry strategies such as partnerships & collaborations and product launches & approvals would drive this market growth. For instance, in November 2022, Minoryx Therapeutics and Neuraxpharm collaborated on concluding the European regulatory process to obtain approval for leriglitazone for the treatment of adult male patients with X-ALD. Leriglitazone is the first approved treatment approved by the European Medicines Agency (EMA.

In May 2022, POXEL SA announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to PXL065 and PXL770 for the treatment of patients with adrenomyeloneuropathy (AMN), the most common form of X-linked adrenoleukodystrophy (ALD).

Restraints

Factors such as the high cost of the treatment, side effects associated with stem cell transplants, a limited number of treatment options, and lack of awareness among people, are expected to hamper the market.

Segment Analysis

The global adrenoleukodystrophy treatment market is segmented based on type, treatment, end users, and region.

The childhood cerebral (cALD) segment accounted for approximately 38.5% of the global adrenoleukodystrophy treatment market share

The childhood cerebral (cALD) segment is expected to hold the largest market share over the forecast period. Childhood cerebral ALD (cALD )is the most devastating form of ALD. X-ALD (X-linked adrenoleukodystrophy) is an orphan neurodegenerative disease. The global incidence of X-ALD is approximately 6-8/100,000 live births. ALD patients, boys, and adult men, at any point in their lifetime, can develop cALD, characterized by demyelinating brain lesions that rapidly progress, leading to acute neurological decline and death.

Moreover, key players in the industry on ongoing advanced research & developments and a rising number of clinical trials in the treatment of adrenoleukodystrophy would drive this segment growth. For instance, in April 2023, Minoryx Therapeutics, announces 24-week interim results from NEXUS. This open-label registration-enabling clinical trial is assessing the safety and efficacy of lead candidate leriglitazone, a novel, brain-penetrant and selective PPAR gamma agonist, to treat pediatric patients with cerebral adrenoleukodystrophy (cALD) - a phenotype of X-linked adrenoleukodystrophy (X-ALD).

Similarly, in November 2023, Minoryx Therapeutics announced that the US Food and Drug Administration (FDA) approved its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (CALD). The CALYX protocol has received both FDA and central IRB approval.

Geographical Analysis

North America accounted for approximately 41.3% of the global adrenoleukodystrophy treatment market share

North America region is expected to hold the largest market share over the forecast period owing to factors such as increasing research and developments, drug approvals, and technological advancements in adrenoleukodystrophy treatment.

Moreover, the rising prevalence of ALD in the U.S. propels this market growth. According to the U.S. National Library of Medicine, in October 2022, the prevalence of ALD is 1 in 20,000 to 50,000 individuals worldwide. In the U.S., 1 in every 21,000 men is thought to be affected by ALD while 1 in 16,800 women are thought to be ALD carriers.

As per a newborn screening conducted in the state of New York, the incidence of ALD at birth was estimated to be 1 in 15,000. Among the 700,000 newborns screened in three years, 45 babies were diagnosed with ALD.

In addition, major players in the industry strategies such as FDA approvals and research & development in the treatment of adrenoleukodystrophy would drive this market growth. For instance, in September 2022, Bluebird Bio received FDA accelerated approval for SKYSONA Gene Therapy for early, active cerebral adrenoleukodystrophy (CALD). This therapy slows down the progression of neurologic dysfunction in boys 4-17 years of age with early and active cerebral adrenoleukodystrophy (CALD).

Also, in January 2022, SwanBio Therapeutics announced that its Investigational New Drug (IND) application for its lead candidate SBT101 for the treatment of adrenomyeloneuropathy (AMN) is cleared by the U.S. Food and Drug Administration (FDA). SBT101 is the first AAV-based gene therapy in development specifically designed for people living with adrenomyeloneuropathy.

Market Segmentation

By Type

• Childhood Cerebral ALD
• Adrenomyeloneuropathy (AMN)
• Addison's Disease

By Treatment

• Stem Cell Transplant
• Corticosteroid Replacement Therapy
• Medications
• Gene Therapy
• Lorenzo's Oil
• Others

By End Users

• Hospitals
• Specialty Clinics
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Italy
  • Spain
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • Rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the adrenoleukodystrophy treatment market include Bluebird Bio Inc., Viking Therapeutics, Magenta Therapeutics, NeuroVia, Minoryx Therapeutics, Poxel S.A., Orpheris, SwanBio Therapeutics, Sperogenix Therapeutics, and Autobahn Therapeutics among others.

Key Developments

• In May 2024, Minoryx Therapeutics announced that the EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended not to grant marketing authorization for NEZGLYAL (leriglitazone) as a treatment for cALD following the completion of the re-examination procedure.
• In February 2024, Neuraxpharm Group and Minoryx Therapeutics, a late-stage biotech company focused on the development of therapies for orphan central nervous system disorders, reaffirm their commitment on Rare Disease Day 2024 to find better treatments for patients suffering from a range of rare diseases, such as leukodystrophy, including X-linked adrenoleukodystrophy (X-ALD) and more specifically cerebral adrenoleukodystrophy (cALD).
• In June 2022, POXEL SA announced its participation at the ULF (United Leukodystrophy Foundation) Scientific Symposium and Family Conference on June at Illinois, USA by using PXL065, two proprietary promising candidates that are focused on the treatment of adrenoleukodystrophy (ALD) which is a rare and genetic disease in adults.

Why Purchase the Report?

• To visualize the global adrenoleukodystrophy treatment market segmentation based on the type, treatment, end users and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of global adrenoleukodystrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global adrenoleukodystrophy treatment market report would provide approximately 62 tables, 55 figures, and 184 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Type
• 3.2. Snippet by Treatment
• 3.3. Snippet by End Users
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing Research & Developments and Rising FDA Approvals of Drugs for Adrenoleukodystrophy Treatment
    • 4.1.1.2. The Increasing Cases of X-linked Recessive Conditions
  • 4.1.2. Restraints
    • 4.1.2.1. High Cost of the Treatment
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's 5 Forces Analysis
• 5.2. Supply Chain Analysis
• 5.3. Unmet Needs
• 5.4. Regulatory Analysis
• 5.5. DMI Opinion

6. By Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 6.1.2. Market Attractiveness Index, By Type
• 6.2. Childhood Cerebral ALD*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Adrenomyeloneuropathy (AMN)
• 6.4. Addison's disease

7. By Treatment

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 7.1.2. Market Attractiveness Index, By Treatment
• 7.2. Stem Cell Transplant
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Corticosteroid Replacement Therapy
• 7.4. Medications
• 7.5. Gene Therapy
• 7.6. Lorenzo's Oil
• 7.7. Others

8. By End Users

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End Users
  • 8.1.2. Market Attractiveness Index, By End Users
• 8.2. Hospitals*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Specialty Clinics
• 8.4. Others

9. By Region

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 9.1.2. Market Attractiveness Index, By Region
• 9.2. North America
  • 9.2.1. Introduction
  • 9.2.2. Key Region-Specific Dynamics
  • 9.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 9.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 9.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users
  • 9.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 9.2.6.1. U.S.
    • 9.2.6.2. Canada
    • 9.2.6.3. Mexico
• 9.3. Europe
  • 9.3.1. Introduction
  • 9.3.2. Key Region-Specific Dynamics
  • 9.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 9.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 9.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users
  • 9.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
  • 9.3.7. Germany
    • 9.3.7.1. U.K.
    • 9.3.7.2. France
    • 9.3.7.3. Italy
    • 9.3.7.4. Spain
    • 9.3.7.5. Rest of Europe
• 9.4. South America
  • 9.4.1. Introduction
  • 9.4.2. Key Region-Specific Dynamics
  • 9.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 9.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 9.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users
  • 9.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
    • 9.4.6.1. Brazil
    • 9.4.6.2. Argentina
    • 9.4.6.3. Rest of South America
• 9.5. Asia Pacific
  • 9.5.1. Introduction
  • 9.5.2. Key Region-Specific Dynamics
  • 9.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 9.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 9.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users
  • 9.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
  • 9.5.7. China
    • 9.5.7.1. India
    • 9.5.7.2. Japan
    • 9.5.7.3. South Korea
    • 9.5.7.4. Rest of Asia Pacific
• 9.6. Middle East and Africa
  • 9.6.1. Introduction
  • 9.6.2. Key Region-Specific Dynamics
  • 9.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
  • 9.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
  • 9.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End Users

10. Competitive Landscape

• 10.1. Competitive Scenario
• 10.2. Product Benchmarking
• 10.3. Company Share Analysis
• 10.4. Key Developments and Strategies

11. Company Profiles

• 11.1. Bluebird Bio Inc.*
  • 11.1.1. Company Overview
  • 11.1.2. Product Portfolio and Description
  • 11.1.3. Financial Overview
  • 11.1.4. Key Developments
• 11.2. Viking Therapeutics
• 11.3. Magenta Therapeutics
• 11.4. NeuroVia
• 11.5. Minoryx Therapeutics
• 11.6. Poxel S.A.
• 11.7. Orpheris
• 11.8. SwanBio Therapeutics
• 11.9. Sperogenix Therapeutics
• 11.10. Autobahn Therapeutics

LIST NOT EXHAUSTIVE

12. Appendix

• 12.1. About Us and Services
• 12.2. Contact Us