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ポンペ病治療の世界市場（2025年～2033年）

Global Pompe Disease Treatment Market - 2025-2033

出版日
ページ情報
英文 159 Pages
納期
即日から翌営業日
カスタマイズ可能
適宜更新あり
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価格表記: USDを日本円(税抜)に換算
本日の銀行送金レート: 1USD=143.57円
ポンペ病治療の世界市場（2025年～2033年）
出版日: 2025年03月11日
発行: DataM Intelligence
ページ情報: 英文 159 Pages
納期: 即日から翌営業日
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概要

世界のポンペ病治療の市場規模は、2024年に13億9,000万米ドルに達し、2033年には21億1,000万米ドルに達する見込みで、2025年～2033年の予測期間中にCAGR4.8％で成長すると予測されています。

ポンペ病は、グリコーゲン貯蔵病II型（GSDII）または酸性マルターゼ欠損症としても知られ、酸性α-グルコシダーゼ（GAA）という酵素の欠損によって引き起こされる、まれな遺伝性の進行性代謝疾患です。この酵素は、物質を消化しリサイクルする細胞の一部であるリソソーム内でグリコーゲン（糖の貯蔵形態）を分解する役割を担っています。GAAが欠損または欠乏すると、リソソーム内、特に骨格筋、心筋、肝臓に存在するリソソーム内にグリコーゲンが異常に蓄積し、進行性の筋力低下、呼吸障害、臓器機能障害を引き起こします。ポンペ病に根本的な治療法はありませんが、進行を遅らせ、生活の質を向上させる治療法はあります。

ポンペ病治療市場は、治療法の進歩、疾患認知度の向上、戦略的な市場開拓などにより、顕著な成長を遂げています。例えば、2021年8月にネクスビアザイム（アバルグルコシダーゼアルファ）などの第2世代ERTが承認され、遅発性ポンペ病の治療効果が向上しました。さらに、2023年9月にFDAはポンビリチ（シパグルコシダーゼ・アルファ-アトガ）とオプフォルダ（ミグルスタット）の併用を承認し、後期発症ポンペ病の成人患者に対する新たな治療選択肢を提供しました。

市場力学：

促進要因と抑制要因

酵素補充療法（ERT）の進歩

酵素補充療法における進歩の高まりは、ポンペ病治療市場の成長を大きく後押ししており、予測期間中も継続的に市場を牽引するとみられます。2021年8月に承認されたネクスビアザイム（アバルグルコシダーゼアルファ）は、ポンペ病治療の大きな進歩を象徴しています。この第2世代の酵素補充療法は、酵素の安定性を改善し、組織への浸透性を向上させることで、特に晩発性ポンペ病（LOPD）に対してより効果的な治療をもたらします。このような有効性の向上により、ネクスビアザイムや類似の治療薬の利用が大幅に増加し、市場の需要を牽引するものと期待されます。

さらに、2023年9月に導入されるポンビリチ（シパグルコシダーゼアルファ-アトガ）とオプフォルダ（ミグルスタット）は、従来のERTで効果が不十分な患者に対する2剤併用療法を提供します。この併用療法は、免疫反応（ERT患者にしばしば起こる）に対処し、成人後期発症ポンペ病の治療成績を改善することを目的としています。新規ERTの承認が進むにつれて、より多くの患者が自分の症状に合った専門的な治療を受けられるようになり、世界的に市場浸透と治療導入率の向上につながります。

大手製薬企業は、ポンペ病に対する次世代ERTの開発に多額の投資を行っています。さらに、複数の企業がポンペ病の先進的な治療法を開発しています。こうした継続的な研究努力が市場全体の成長を後押しすると期待されています。

承認済みの治療薬が限られていることが市場成長を抑制する可能性

承認済みの治療薬が限られていることは、治療へのアクセスに障壁をもたらし、アンメット・メディカル・ニーズを高め、患者の選択肢を狭めているため、ポンペ病治療市場の成長を大きく妨げています。現在のところ、FDAが承認したポンペ病治療薬は、マイオザイム、ルミザイム、ネクスビアザイム、ポンビリチ＋オプフォルダなどの酵素補充療法（ERT）を中心に数種類しかありません。これらの治療は命を救うものではありますが、根治療法ではありません。症状の管理や病気の進行を遅らせることには役立ちますが、病気による障害を止めたり、元に戻したりすることはできません。

根治的な治療法（臨床試験中の遺伝子治療など）がないため、患者は生涯ERTに依存することになりますが、特に酵素に対する免疫反応を起こす患者や、組織への浸透に限界がある患者にとっては、必ずしも完全な緩和が得られるとは限りません。手頃な価格の治療薬が限られているため、特にヘルスケアのインフラがしっかりしていない地域では、アクセスが制限されています。多くの新興諸国では、たとえ治療法が承認されても、経済的制約や償還制度の欠如により、広く利用されることはありません。

新治療法の承認プロセスは遅々として進まず、潜在的な治療法の研究は続けられてはいますが、過去10年間に承認された新薬はほとんどありません。2023年現在、遺伝子治療や酵素最適化戦略など、臨床試験段階にある有望な治療法がいくつかある一方で、ほとんどの治療法はまだ承認されていません。市場は、ERT以外の治療選択肢の多様性の欠如に直面しており、ERTに反応しない患者には代替選択肢がほとんどないため、成長の妨げとなっています。このように承認のペースが遅いため、潜在的な市場規模や新しい治療法の採用が大幅に制限されています。

目次

第1章 市場イントロダクションと範囲

  • レポートの目的
  • 調査のカバー範囲と定義
  • 調査の対象範囲

第2章 エグゼクティブの洞察と重要なポイント

  • 市場のハイライトと戦略的ポイント
  • 主な動向と将来の予測
  • スニペット：疾病タイプ
  • スニペット：薬剤別
  • スニペット：投与経路別
  • スニペット：年齢層別
  • スニペット：地域別

第3章 市場力学

  • 影響要因
    • 促進要因
      • 酵素補充療法（ERT）の進歩
    • 抑制要因
      • 限られた承認済みの治療薬
    • 機会
    • 影響分析

第4章 戦略的洞察と業界展望

  • 市場のリーダーと先駆者
    • 新たな先駆者と著名企業
    • 最大の販売ブランドを持つ確立されたリーダー
    • 確立された製品を持つ市場リーダー
  • CXOの視点
  • 最新の開発とブレークスルー
  • ケーススタディ/進行中の研究
  • 規制と償還の情勢
    • 北米
    • 欧州
    • アジア太平洋
    • ラテンアメリカ
    • 中東・アフリカ
  • 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
  • パイプライン分析
  • 疫学分析
  • 特許分析
  • SWOT分析
  • アンメットニーズとギャップ
  • 市場参入と拡大のための推奨戦略
  • シナリオ分析ベストケース、ベースケース、ワーストケースの予測
  • 価格分析と価格市場力学
  • キーオピニオンリーダー

第5章 ポンペ病治療市場：疾病タイプ別

  • 乳児期発症ポンペ病
    • 典型的幼児型
    • 非典型的乳児型
  • 遅発性ポンペ病

第6章 ポンペ病治療市場：薬剤別

  • アルグルコシダーゼアルファ
  • アバルグルコシダーゼアルファ
  • シパグルコシダーゼアルファ
  • ミグルスタット

第7章 ポンペ病治療市場：投与経路別

  • 非経口
  • 経口

第8章 ポンペ病治療市場：年齢層別

  • 小児
  • 大人

第9章 ポンペ病治療市場：地域別の市場分析と成長機会

  • 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
  • 中東・アフリカ

第10章 競合情勢と市場ポジショニング

  • 競合状況の概要と主要な市場企業
  • 市場シェア分析とポジショニングマトリックス
  • 戦略的パートナーシップ、合併、買収
  • 製品ポートフォリオとイノベーションにおける主な発展
  • 企業ベンチマーク

第11章 企業プロファイル

  • Sanofi
    • 会社概要
    • 製品ポートフォリオと概要
    • 財務概要
    • 主な発展
    • SWOT分析
  • Amicus Therapeutics
  • AskBio Inc.
  • Beijing GeneCradle Technology Co., Ltd.
  • Aro Biotherapeutics Company

第12章 前提条件と調査手法

  • データ収集方法
  • データの三角測量
  • 予測技術
  • データの検証とバリデーション

第13章 付録

目次
Product Code: PH9210

The global Pompe disease treatment market reached US$ 1.39 billion in 2024 and is expected to reach US$ 2.11 billion by 2033, growing at a CAGR of 4.8% during the forecast period of 2025-2033.

Pompe disease, also known as Glycogen Storage Disease Type II (GSDII) or Acid Maltase Deficiency, is a rare, inherited, and progressive metabolic disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). This enzyme is responsible for breaking down glycogen (a stored form of sugar) within lysosomes, a part of the cell that digests and recycles substances. When GAA is deficient or absent, glycogen accumulates abnormally within lysosomes, particularly in those present in skeletal muscles, heart muscles, and the liver, leading to progressive muscle weakness, respiratory issues, and organ dysfunction. There is no cure for Pompe disease, but treatments can slow its progression and improve the quality of life.

The Pompe disease treatment market is experiencing notable growth, driven by advancements in therapies, increased disease awareness, and strategic industry developments. For instance, the approval of second-generation ERTs, such as Nexviazyme (avalglucosidase alfa) in August 2021, has enhanced treatment efficacy for late-onset Pompe disease. Additionally, in September 2023, the FDA approved Pombiliti (cipaglucosidase alfa-atga) in combination with Opfolda (miglustat), offering a new therapeutic option for adults with late-onset Pompe disease.

Market Dynamics: Drivers & Restraints

Advancements in Enzyme Replacement Therapy (ERT)

The rising advancements in enzyme replacement therapy are significantly driving the growth of the Pompe disease treatment market and are expected to continuously drive the market over the forecast period. Nexviazyme (avalglucosidase alfa), approved in August 2021, represents a major advancement in Pompe disease treatment. This second-generation enzyme replacement therapy offers improved enzyme stability and better tissue penetration, resulting in more effective treatment, particularly for late-onset Pompe disease (LOPD). This efficacy boost is expected to significantly increase the uptake of Nexviazyme and similar therapies, driving market demand.

Additionally, the introduction of Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) in September 2023 provides a two-component therapy for patients who have not responded well to conventional ERT. This combination therapy addresses immune responses (which often occur in ERT patients) and aims to improve treatment outcomes in adults with late-onset Pompe disease. With increasing approvals of novel ERTs, more patients can access specialized therapies suited to their condition, leading to wider market penetration and higher treatment adoption rates globally.

Major pharmaceutical companies are investing heavily in the development of next-generation ERTs for Pompe disease. Additionally, several companies are developing advanced therapies for Pompe disease. These continued research efforts are expected to boost the overall market growth.

Below are the key pipeline drugs and their current phase of clinical development.

Limited Availability of Approved Therapies May Restrain the Market Growth.

The limited availability of approved therapies is significantly hampering the growth of the Pompe disease treatment market, as it creates barriers to treatment access, increases unmet medical needs, and limits options for patients. As of now, there are only a few FDA-approved therapies for Pompe disease, primarily enzyme replacement therapies (ERT) like Myozyme, Lumizyme, Nexviazyme, and Pombiliti + Opfolda. While these treatments are life-saving, they are not curative. They help manage symptoms and slow disease progression, but they do not stop or reverse the damage caused by the disease, especially in late-onset cases.

The lack of curative options (like gene therapy, which is still in clinical trials) means patients are dependent on lifelong ERT, which doesn't always provide complete relief, especially for those who develop immune responses to the enzymes or for those who experience limited efficacy in tissue penetration. The limited availability of affordable therapies restricts access, particularly in regions with less robust healthcare infrastructure. In many developing countries, even if the therapies are approved, they are not widely available due to economic constraints and lack of reimbursement programs.

The regulatory process for new treatments is slow, and while there is ongoing research into potential therapies, very few new drugs have been approved in the last decade. As of 2023, while there are several promising treatments in the clinical trial phase, such as gene therapies and enzyme optimization strategies, most therapies are still not approved for widespread use. The market faces a lack of diversity in treatment options beyond ERT, which hampers growth because patients who do not respond well to ERTs are left with few alternative options. This slow pace of approval significantly limits the potential market size and adoption of new therapies.

Segment Analysis

The global Pompe disease treatment market is segmented based on disease type, Drug, route of administration, Age Group, and region.

Product Type:

The enzyme replacement therapy in the Drug segment is expected to dominate with a higher market share.

Myozyme (alglucosidase alfa), the first FDA-approved ERT for Pompe disease, was introduced in 2006 and quickly became the gold standard for treating infantile-onset Pompe disease (IOPD). Since its approval, it has been widely adopted and continues to be the primary treatment option. Later, Lumizyme, an alternative formulation of Myozyme, was introduced to treat late-onset Pompe disease (LOPD). Both drugs provide essential enzyme replacement for patients who lack the enzyme acid alpha-glucosidase (GAA), helping to manage symptoms and slow disease progression.

The approval of Nexviazyme (avalglucosidase alfa) in 2021 expanded the range of enzyme replacement therapies available to patients. Nexviazyme, specifically designed for late-onset Pompe disease, is considered more efficient at clearing glycogen from tissues compared to earlier treatments like Myozyme, improving clinical outcomes. Pombiliti (cipaglucosidase alfa) combined with Opfolda (miglustat) was approved in 2023, offering a new therapy for patients who don't respond well to standard ERT, particularly in adult LOPD patients. This combination therapy presents a broader solution within the ERT segment.

Enzyme replacement therapy has been shown to significantly improve the quality of life and clinical outcomes for patients, including reducing muscle weakness, improving cardiopulmonary function, and increasing mobility. Clinical trials of Nexviazyme have demonstrated a significant improvement in muscle strength, respiratory function, and motor skills, leading to its adoption as an alternative treatment for late-onset cases. Myozyme and Lumizyme have also helped patients with infantile-onset Pompe disease survive longer, with some infants living past their first year, where untreated Pompe disease would typically lead to early death due to heart and respiratory failure.

Geographical Analysis

North America is expected to hold a significant position in the Pompe disease treatment market share

North America, especially the United States, has one of the highest Pompe disease rates globally, significantly driving the demand for effective treatment solutions. For instance, according to the Cleveland Clinic Organization, in the United States, Pompe disease affects about 1 in every 40,000 people. North America is a leader in the approval and commercialization of Enzyme Replacement Therapies (ERTs), including Myozyme, Lumizyme, Nexviazyme, and Pombiliti. The U.S. Food and Drug Administration (FDA) is often the first regulatory body to approve new treatments for Pompe disease.

For instance, in September 2023, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD), weighing >=40 kg, and who are not improving on their current enzyme replacement therapy (ERT).

North America, particularly the United States, is home to leading pharmaceutical companies and research institutions actively developing novel therapies for Pompe disease. Companies like Sanofi Genzyme, Amicus Therapeutics, and Aro Biotherapeutics have invested significantly in clinical trials, aiming to improve existing therapies or develop new treatment modalities like gene therapy and chaperone therapy.

For instance, in February 2025, Aro Biotherapeutics announced that it had received U.S. Food and Drug Administration (FDA) clearance of the Investigational New Drug Application (IND) for ABX1100, a novel therapy currently being investigated in a phase 1 study in late-onset Pompe disease (LOPD) patients.

Asia-Pacific is growing at the fastest pace in the Pompe disease treatment market

Asia-Pacific countries have been improving their regulatory frameworks to speed up the approval of innovative treatments. For instance, the China National Medical Products Administration (NMPA) and Japan's Pharmaceuticals and Medical Devices Agency (PMDA) are actively working to expedite the approval processes for new drugs, including those for rare diseases like Pompe disease.

For instance, according to the National Institute of Health, Alglucosidase alfa received marketing approval for the treatment of Pompe disease in Japan in 2007. Additionally, Japan has been at the forefront of accelerated approval processes for rare diseases, including Pompe disease treatments like Myozyme and Nexviazyme, which are now widely available in the country. Japan's focus on rare diseases and its advanced regulatory processes have contributed to the high adoption of approved therapies in the region.

The Asia-Pacific region, particularly countries like China, India, Japan, and South Korea, are leading the charge in providing better access to enzyme replacement therapies (ERTs) and improving the overall healthcare framework for rare diseases. With a strong focus on early diagnosis, improving patient access, and growing R&D investments, the APAC region is poised for significant growth in the coming years.

Competitive Landscape

Why Purchase the Report?

  • Pipeline & Innovations: Reviews ongoing clinical trials, product pipelines, and forecasts upcoming advancements in medical devices and pharmaceuticals.
  • Product Performance & Market Positioning: Analyze product performance, market positioning, and growth potential to optimize strategies.
  • Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
  • Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
  • Market Updates & Industry Changes: This covers recent regulatory changes, new policies, and emerging technologies.
  • Competitive Strategies: Analyze competitor strategies, market share, and emerging players.
  • Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
  • Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
  • Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
  • Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient product delivery.
  • Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
  • Post-market Surveillance: Uses post-market data to enhance product safety and access.
  • Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The global Pompe disease treatment market report delivers a detailed analysis with 55 key tables, more than 61 visually impactful figures, and 159 pages of expert insights, providing a complete view of the market landscape.

Target Audience 2024

  • Manufacturers: Pharmaceutical, Medical Device, Biotech Companies, Contract Manufacturers, Distributors, hospitals.
  • Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
  • Technology & Innovation: AI/Robotics Providers, R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
  • Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
  • Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
  • Supply Chain: Distribution and Supply Chain Managers.
  • Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
  • Academic & Research: Academic Institutions.

Table of Contents

1. Market Introduction and Scope

  • 1.1. Objectives of the Report
  • 1.2. Report Coverage & Definitions
  • 1.3. Report Scope

2. Executive Insights and Key Takeaways

  • 2.1. Market Highlights and Strategic Takeaways
  • 2.2. Key Trends and Future Projections
  • 2.3. Snippet by Disease Type
  • 2.4. Snippet by Drug
  • 2.5. Snippet by Route of Administration
  • 2.6. Snippet by Age Group
  • 2.7. Snippet by Region

3. Dynamics

  • 3.1. Impacting Factors
    • 3.1.1. Drivers
      • 3.1.1.1. Advancements in Enzyme Replacement Therapy (ERT)
    • 3.1.2. Restraints
      • 3.1.2.1. Limited Availability of Approved Therapies
    • 3.1.3. Opportunity
    • 3.1.4. Impact Analysis

4. Strategic Insights and Industry Outlook

  • 4.1. Market Leaders and Pioneers
    • 4.1.1. Emerging Pioneers and Prominent Players
    • 4.1.2. Established leaders with largest selling Brand
    • 4.1.3. Market leaders with established Product
  • 4.2. CXO Perspectives
  • 4.3. Latest Developments and Breakthroughs
  • 4.4. Case Studies/Ongoing Research
  • 4.5. Regulatory and Reimbursement Landscape
    • 4.5.1. North America
    • 4.5.2. Europe
    • 4.5.3. Asia Pacific
    • 4.5.4. Latin America
    • 4.5.5. Middle East & Africa
  • 4.6. Porter's Five Forces Analysis
  • 4.7. Supply Chain Analysis
  • 4.8. Pipeline Analysis
  • 4.9. Epidemiology Analysis
  • 4.10. Patent Analysis
  • 4.11. SWOT Analysis
  • 4.12. Unmet Needs and Gaps
  • 4.13. Recommended Strategies for Market Entry and Expansion
  • 4.14. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
  • 4.15. Pricing Analysis and Price Dynamics
  • 4.16. Key Opinion Leaders

5. Pompe Disease Treatment Market, By Disease Type

  • 5.1. Introduction
    • 5.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Disease Type
    • 5.1.2. Market Attractiveness Index By Disease Type
  • 5.2. Infantile-Onset Pompe Disease*
    • 5.2.1. Introduction
    • 5.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
    • 5.2.3. Classic Infantile Form
    • 5.2.4. Non-Classic Infantile Form
  • 5.3. Late-Onset Pompe Disease

6. Pompe Disease Treatment Market, By Drug

  • 6.1. Introduction
    • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 6.1.2. Market Attractiveness Index, By Drug
  • 6.2. Alglucosidase Alfa*
    • 6.2.1. Introduction
    • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 6.3. Avalglucosidase Alfa
  • 6.4. Cipaglucosidase Alfa
  • 6.5. Miglustat

7. Pompe Disease Treatment Market, 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. Parenteral*
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 7.3. Oral

8. Pompe Disease Treatment Market, By Age Group

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 8.1.2. Market Attractiveness Index By Age Group
  • 8.2. Pediatrics*
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3. Adults

9. Pompe Disease Treatment Market, By Regional Market Analysis and Growth Opportunities

  • 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 Disease Type
    • 9.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 9.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.2.7.1. U.S.
      • 9.2.7.2. Canada
      • 9.2.7.3. 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 Disease Type
    • 9.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 9.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.3.7.1. Germany
      • 9.3.7.2. U.K.
      • 9.3.7.3. France
      • 9.3.7.4. Spain
      • 9.3.7.5. Italy
      • 9.3.7.6. 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 Disease Type
    • 9.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 9.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.4.7.1. Brazil
      • 9.4.7.2. Argentina
      • 9.4.7.3. 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 Disease Type
    • 9.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 9.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group
    • 9.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
      • 9.5.7.1. China
      • 9.5.7.2. India
      • 9.5.7.3. Japan
      • 9.5.7.4. South Korea
      • 9.5.7.5. 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 Disease Type
    • 9.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug
    • 9.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
    • 9.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Age Group

10. Competitive Landscape and Market Positioning

  • 10.1. Competitive Overview and Key Market Players
  • 10.2. Market Share Analysis and Positioning Matrix
  • 10.3. Strategic Partnerships, Mergers, & Acquisitions
  • 10.4. Key Developments in Product Portfolios and Innovations
  • 10.5. Company Benchmarking

11. Company Profiles

  • 11.1. Sanofi*
    • 11.1.1. Company Overview
    • 11.1.2. Product Portfolio and Description
    • 11.1.3. Financial Overview
    • 11.1.4. Key Developments
    • 11.1.5. SWOT Analysis
  • 11.2. Amicus Therapeutics
  • 11.3. AskBio Inc.
  • 11.4. Beijing GeneCradle Technology Co., Ltd.
  • 11.5. Aro Biotherapeutics Company

LIST NOT EXHAUSTIVE

12. Assumption and Research Methodology

  • 12.1. Data Collection Methods
  • 12.2. Data Triangulation
  • 12.3. Forecasting Techniques
  • 12.4. Data Verification and Validation

13. Appendix

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