希少神経疾患治療の世界市場-2024-2031

概要

世界の希少神経疾患治療市場は、2023年に1,394億米ドルに達し、2031年には2,697億米ドルに達すると予測され、予測期間2024-2031年のCAGRは8.6%です。

希少神経疾患は、希少神経系疾患または極めて稀な神経疾患とも呼ばれ、神経系に影響を及ぼす様々な珍しい疾患です。希少神経疾患は、主に中枢神経系と末梢神経系、および筋肉に影響を及ぼす異種の疾患群です。これらの疾患は、有病率が低いため「まれ」とみなされ、一般的には2,000人に1人未満が罹患すると定義されています。

人体の神経系を対象とするまれな神経疾患には、脳と脊髄が含まれます。アイカルディ症候群、アイカルディ・グティエール症候群、反射性交感神経性ジストロフィー症候群、バターリャ・ネリ症候群、クロイツフェルト・ヤコブ病、アグノシアなど、さまざまな希少神経疾患があります。

希少神経疾患治療には、認知行動療法、対人関係心理療法、サイバーナイフ（定位放射線治療）などさまざまな治療法があります。レベチラセタム（ケプラ）、トピラマート（トパマックス）、ラモトリギン（ラミクタール）、オクスカルバゼピン（トリレプタル）、ジバルプロエクスナトリウム（デパコート）などの薬剤があります。

市場力学：

促進要因

希少神経疾患の有病率の増加

世界の希少神経疾患治療市場の需要は、複数の要因によって牽引されています。主な要因の一つは、希少神経疾患の有病率の増加です。世界保健機関（WHO）によると、2024年5月には、3人に1人以上が神経疾患に罹患しており、世界中で病気や障害の主な原因となっています。

希少な神経疾患には、筋萎縮性側索硬化症（ALS）、デュシェンヌ型筋ジストロフィー、垂直注視性麻痺、ハンチントン病などがあります。2023年11月に発表されたOrphanetの報告によると、慢性炎症性脱髄性多発神経炎の有病率は、欧州では10万人当たり3.7人であり、ALSの有病率は2023年において世界で3.85人、欧州連合（EU）で5.2人です。同じ出典によると、ハンチントン病の有病率は2023年にEUで12.0です。

さらに、提携・共同研究、臨床試験・調査研究の増加、製品の上市・承認といった主要企業の戦略は、市場の成長を促進すると予想されます。例えば、2023年7月にBiogen Inc.は、バイオジェンがリータを1株当たり現金172.50ドル（企業価値約73億米ドル）で買収することで合意したと発表しました。

また、2023年4月、国際的な希少疾病用医薬品の開発・商業化企業であるオルファランSAは、米国でCuvriorの商業的発売を発表しました。これは新規トリエンチン四塩酸塩（TETA-4HCl）です。D-ペニシラミンに脱抑制・寛容な安定型ウィルソン病成人患者の治療に使用できるようになった。

同様に2023年4月、バイオジェン社は、スーパーオキシドジスムターゼ1（SOD1）遺伝子に変異を有する成人の筋萎縮性側索硬化症（ALS）の治療薬として、QALSODY（tofersen）100mg/15mL注射剤が米国食品医薬品局（FDA）に承認されたと発表しました。

制約事項

高額な治療費、厳しい規制要件、認識・診断の不足、患者数の少なさ、限られた治療オプション、償還の課題、熟練・訓練されたヘルスケア専門家の不足などの課題が市場の妨げになると予想されます。

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目次

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
    • 希少神経疾患の有病率の増加
    • 希少神経疾患に対する新薬の革新的開発
  • 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
• 価格分析
• 規制分析

第6章 薬剤タイプ別

• 生物製剤
• 低分子医薬品

第7章 投与経路別

• 静脈注射
• 経口

第8章 適応症別

• ドラベ症候群
• 副腎白質ジストロフィー
• ナルコレプシー
• アンジェルマン症候群
• 筋萎縮性側索硬化症
• その他

第9章 流通チャネル別

• 病院薬局
• 小売薬局
• オンライン薬局

第10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
  • スペイン
  • イタリア
  • 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地域
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• Novartis AG
  • 会社概要
  • 製品ポートフォリオと説明
  • 財務概要
  • 主な発展
• Pfizer, Inc
• Johnson & Johnson Services, Inc
• US WorldMeds LLC（Solstice Neurosciences LLC）
• Aquestive Therapeutics Inc
• Sanofi S.A.
• Merck & Co., Inc
• CSL Ltd
• Merz Pharma GmbH & Co. KGaA
• Kedrion Biopharma Inc

第13章 付録

Overview

The global rare neurological disease drugs market reached US$ 139.4 billion in 2023 and is expected to reach US$ 269.7 billion by 2031 growing with a CAGR of 8.6% during the forecast period 2024-2031.

Rare neurological diseases, also referred to as rare neurologic disorders or orphan neurological disorders, are a range of unusual and varied conditions that impact the nervous system. Rare neurological diseases are a heterogeneous group of disorders that primarily affect the central and peripheral nervous systems, as well as the muscles. These conditions are considered "rare" due to their low prevalence, typically defined as affecting less than 1 in 2,000 people.

Rare neurological diseases that target the nervous system of the human body include the brain and spinal cord. There are various rare neurological disorders such as Aicardi syndrome, Aicardi-Goutieres syndrome, Reflex Sympathetic Dystrophy Syndrome, Battaglia-Neri syndrome, Creutzfeldt Jakob Disease, Agnosia, and many others.

There are various therapies used for the treatment of rare neurological diseases drugs such as cognitive behavioral therapy, interpersonal psychotherapy, cyberknife, and others. Drugs such as levetiracetam (Keppra), topiramate (Topamax), lamotrigine (Lamictal), oxcarbazepine (Trileptal), and divalproex sodium (Depakote).

Market Dynamics: Drivers

Increasing Prevalence of Rare Neurological Diseases

The demand for the global rare neurological disease drugs market is driven by multiple factors. One of the primary factors is the increasing prevalence of rare neurological diseases. According to the World Health Organization (WHO), in May 2024, over 1 in 3 people were affected by neurological conditions, the leading cause of illness and disability worldwide.

Some rare neurological diseases include amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, Vertical gaze palsy, and Huntington's disease, among others. As per the Orphanet report published in November 2023, the prevalence of chronic inflammatory demyelinating polyneuropathy is 3.7 per 100,000 individuals in Europe, and the prevalence of ALS is 3.85 worldwide and 5.2 in the European Union (EU) in 2023. As per the same source, the prevalence of Huntington's disease is 12.0 in the EU in 2023.

Furthermore, key players' strategies such as partnerships & collaborations, a rising number of clinical trials, research studies, and product launches & approvals are expected to propel the market growth. For instance, in July 2023, Biogen Inc. Reata Pharmaceuticals, Inc. announced the companies had entered into a definitive agreement under which Biogen has agreed to acquire Reata for $172.50 per share in cash, reflecting an enterprise value of approximately $7.3 billion.

Also, in April 2023, Orphalan SA, an international orphan drug development and commercialization company, announced the commercial launch in the U.S. for Cuvrior. It is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.

Similarly, in April 2023, Biogen Inc announced that the U.S. Food and Drug Administration (FDA) has approved QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

Restraints

Factors such as the high cost of treatment, stringent regulatory requirements, lack of awareness & diagnosis, small patient population, limited treatment options, reimbursement challenges, and lack of skilled & trained healthcare professionals are expected to hamper the market.

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Segment Analysis

The global rare neurological disease drugs are segmented based on drug type, route of administration, application, distribution channel, and region.

The small molecules segment accounted for approximately 64.3% of the global rare neurological disease drugs market share

The small molecules segment is expected to hold the largest market share over the forecast period. Small molecule drugs have several advantages that make them well-suited for rare neurological disease treatments. Their small size allows them to easily penetrate the blood-brain barrier and access intracellular targets within the central nervous system.

This enables the modulation of specific protein functions implicated in rare neurological disorders. Additionally, small molecules can be manufactured more quickly and cost-effectively compared to larger biological drugs.

Moreover, key players in the industry strategies such as partnerships & collaborations, product approvals, and financial investments would drive this market growth. For instance, in April 2024, Ipsen and Skyhawk Therapeutics announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases.

Similarly, in January 2024, Biogen Inc. announced plans to reprioritize its resources in Alzheimer's disease (AD), a strategic therapeutic area expected to drive near and long-term growth. The company will continue to advance LEQEMBI (lecanemab-irmb), the first anti-amyloid beta treatment with FDA traditional approval in the United States, and will accelerate the development of potential new treatment modalities, including its ASO targeting tau (BIIB080) and an oral small molecule inhibitor of tau aggregation (BIIB113).

Also, in March 2023, Rapport Therapeutics launched with $100 million in a series A financing to support its novel platform to discover precision-targeted small molecule drugs to treat neurological disorders.

Geographical Analysis

North America accounted for approximately 44.3% of the global rare neurological disease drugs market share

North American region is expected to hold the largest market share over the forecast period owing to the growing incidence of rare neurological disorders, availability of reimbursement for the treatment of rare diseases, and increasing research and development (R&D) in the region.

As fda.gov stated in February 2023, a rare disease is any disease that affects fewer than 200,000 people in the U.S. Approximately 25 to 30 million Americans are living with a rare disease (about 1 in 10 people), and many rare diseases have few or no available treatment options.

Moreover, a major number of key players' presence, well-advanced healthcare infrastructure, and government initiatives such as awareness programs would drive this market growth. For instance, in June 2024, Neurogene Inc. announced that its NGN-401 gene therapy for Rett syndrome has been selected to participate in the U.S. Food and Drug Administration (FDA) Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.

Similarly, in May 2022, CDER launched the Accelerating Rare Disease Cures (ARC) Program. The ARC Program seeks to harness CDER's collective expertise and activities to drive scientific and regulatory innovation for rare diseases.

In addition, the rising number of clinical trials, product launched and approvals would propel this market growth in this region. For instance, in March 2023, The FDA signed off on Reata's Skyclarys (omoveloxolone) for the treatment of Friedreich's ataxia (FA), a hereditary neurological disorder that strikes during adolescence and leaves patients wheelchair-bound and often cuts short their lives. Skyclarys became the first drug approved for the condition, which affects roughly 5,000 who have been diagnosed in the United States and 22,000 worldwide.

Also, in September 2022, Acadia Pharmaceuticals announced Trofinetide's new drug application for the treatment of Rett syndrome has been accepted for filing and review by the U.S. FDA

Similarly, in June 2024, Lucy Therapeutics announced it has raised $12.5m in additional funding to develop potentially new drugs and advance the company's research programs for neurological diseases.

Market Segmentation

By Drug Type

• Biologics
• Small Molecules

By Route of Administration

• Intravenous
• Oral

By Application

• Dravet syndrome
• Adrenoleukodystrophy
• Narcolepsy
• Angelman syndrome
• Amyotrophic Lateral Sclerosis
• Others

By Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • 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 rare neurological disease drugs market include Novartis AG, Pfizer, Inc., Johnson & Johnson Services, Inc., US WorldMeds LLC (Solstice Neurosciences LLC), Aquestive Therapeutics Inc., Sanofi S.A., Merck & Co., Inc., CSL Ltd, Merz Pharma GmbH & Co. KGaA, and Kedrion Biopharma Inc. among others.

Key Developments

• In May 2024, Biogen Inc announced the European Commission (EC) has granted marketing authorization under exceptional circumstances and maintained orphan designation for QALSODY (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene (SOD1-ALS).
• In April 2024, NS Pharma Announces Research Alliance with MiNA Therapeutics to Develop Therapies for Rare Diseases of the Central Nervous System.
• In February 2024, Roche Pharma India announced the launch of its blockbuster breakthrough drug, Ocrevus (Ocrelizumab), for the treatment of multiple sclerosis (MS), expanding its neurology portfolio to cater to unmet needs of numerous patients grappling with this debilitating disease in India
• In September 2023, Harmony Biosciences Holdings, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisantf for the treatment of idiopathic hypersomnia (IH).

Why Purchase the Report?

• To visualize the global rare neurological disease drugs market segmentation based on drug type, route of administration, application, distribution channel, 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 rare neurological disease 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 rare neurological disease drugs market report would provide approximately 69 tables, 68 figures, and 183 pages.

Target Audience 2023

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

Table of Contents

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 Drug Type
• 3.2. Snippet by Route of Administration
• 3.3. Snippet by Application
• 3.4. Snippet by Distribution Channel
• 3.5. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing Prevalence of Rare Neurological Diseases
    • 4.1.1.2. Innovative Development of New Drugs for Rare Neurological Diseases
  • 4.1.2. Restraints
    • 4.1.2.1. High Cost of Treatment
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis

6. By Drug Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 6.1.2. Market Attractiveness Index, By Drug Type
• 6.2. Biologics
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Small Molecules

7. By Route of Administration

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 7.1.2. Market Attractiveness Index, By Route of Administration
• 7.2. Intravenous*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Oral

8. By Application

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 8.1.2. Market Attractiveness Index, By Application
• 8.2. Dravet syndrome *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Adrenoleukodystrophy
• 8.4. Narcolepsy
• 8.5. Angelman syndrome
• 8.6. Amyotrophic Lateral Sclerosis
• 8.7. Others

9. By Distribution Channel

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 9.1.2. Market Attractiveness Index, By Distribution Channel
• 9.2. Hospital Pharmacies*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Retail Pharmacies
• 9.4. Online Pharmacies

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.7.1. U.S.
    • 10.2.7.2. Canada
    • 10.2.7.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.7.1. Germany
    • 10.3.7.2. U.K.
    • 10.3.7.3. France
    • 10.3.7.4. Spain
    • 10.3.7.5. Italy
    • 10.3.7.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.7.1. Brazil
    • 10.4.7.2. Argentina
    • 10.4.7.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
  • 10.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.7.1. China
    • 10.5.7.2. India
    • 10.5.7.3. Japan
    • 10.5.7.4. South Korea
    • 10.5.7.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Novartis AG*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Pfizer, Inc
• 12.3. Johnson & Johnson Services, Inc
• 12.4. US WorldMeds LLC (Solstice Neurosciences LLC)
• 12.5. Aquestive Therapeutics Inc
• 12.6. Sanofi S.A.
• 12.7. Merck & Co., Inc
• 12.8. CSL Ltd
• 12.9. Merz Pharma GmbH & Co. KGaA
• 12.10. Kedrion Biopharma Inc

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us