ゴーシェ病治療の世界市場（2025年～2033年）

世界のゴーシェ病治療の市場規模は、2024年に17億6,000万米ドルに達し、2033年には22億8,000万米ドルに達すると予測され、予測期間2025年～2033年のCAGRは2.9%で成長する見込みです。

ゴーシェ病は、グルコセレブロシダーゼ（グルコシルセラミダーゼとしても知られる）と呼ばれる酵素の欠損によって引き起こされるまれな遺伝性代謝疾患であり、主に脾臓、肝臓、骨、骨髄など、体のさまざまな臓器にグルコセレブロシドと呼ばれる脂肪物質が蓄積します。この蓄積は、ゴーシェ病のタイプや罹患する臓器によって重症度が異なる様々な症状を引き起こします。

ゴーシェ病治療酵素補充療法（ERT）や基質減少療法（SRT）などの選択肢は、生活の質を著しく改善させますが、現在のところ治療法はありません。遺伝子治療が長期的な解決策となりうる可能性が出てきていますが、まだ開発途上です。現在進行中の遺伝子治療の研究と治療の進歩に注目している企業は有望です。

例えば、2024年7月、スパー・セラピューティクスは、ゴーシェ病に対する遺伝子治療候補が、第I/II相試験で良好な初期データを得た後、第III相試験に移行すると発表しました。GALILEO-1試験は、ゴーシェ病の治療薬としてアデノ随伴ウイルス（AAV）遺伝子治療候補であるFLT201を研究しています。FLT-201はグルコシルセラミダーゼβ（GBA1）遺伝子活性化因子として作用します。この遺伝子の変異はゴーシェ病の原因となります。この薬剤は、欠損したGBA1遺伝子の機能を回復させ、病状を緩和します。

市場力学：

促進要因と抑制要因

酵素補充療法と基質低減療法の拡大

酵素補充療法と基質低減療法の拡大は、ゴーシェ病治療市場の成長を大きく後押ししており、予測期間中も市場を牽引するとみられます。酵素補充療法は、1型ゴーシェ病の最も一般的で確立された治療法です。これは、ゴーシェ病患者に欠損している酵素である合成グルコセレブロシダーゼを静脈内投与するもので、グルコセレブロシドの分解を助け、肝臓、脾臓、骨髄などの臓器への蓄積を防ぎます。

例えば、FDAによって承認された最初のERT薬であるイミグルセラーゼ（セレザイム）は、依然としてゴールドスタンダードであり、ベラグルセラーゼ・アルファ（Vpriv）やタリグルセラーゼ・アルファ（Elelyso）のような他の薬も市場で人気を集めています。これらの酵素補充療法は、ゴーシェ患者の臓器肥大（脾臓や肝臓の肥大）や骨関連合併症の治療に大きな有効性を示しています。

基質低減療法（SRT）は酵素補充療法の代替療法あるいは補助療法であり、特に点滴よりも内服を好む患者や酵素補充療法に耐えられない患者にとって有効です。SRTは、ゴーシェ病で蓄積する基質であるグルコセレブロシドの産生を減少させることで効果を発揮します。例えば、エリグルスタット（セルデルガ）やミグルスタット（ザベスカ）などがゴーシェ病で承認されている主な基質減少療法です。これらの薬剤は経口投与が可能で、侵襲性の高い静脈内ERTに比べ患者のコンプライアンスが向上します。

全体として、ERTとSRTの拡大はゴーシェ病治療市場の成長に重要な役割を果たしています。これらの治療法は患者の予後を改善するだけでなく、世界的に治療へのアクセスを向上させています。特に経口療法（SRT）は利便性が高く、患者の治療へのアドヒアランスを高めるのに役立っています。

副作用と長い治療期間

ゴーシェ病の治療、特に酵素補充療法（ERT）と基質除去療法（SRT）に伴う副作用と長い治療期間は、ゴーシェ病治療市場の成長を妨げる大きな障壁となっています。これらの治療法は疾患の管理には極めて重要ですが、患者のコンプライアンス、治療の普及、市場全体の拡大に影響を及ぼす可能性のある課題があります。

ERTは効果的ではありますが、グルコセレブロシダーゼ（セレザイム、Vpriv、エレリッソなど）の点滴静注を伴うため、患者のアドヒアランスや全体的な治療効果に影響を及ぼす可能性のある副作用がいくつかあります。一般的な副作用には、発熱、悪寒、頭痛、吐き気などがあり、多くの場合、点滴中または点滴後に起こります。これらの反応は不快感の原因となり、治療継続をためらう患者もいます。酵素に対するアレルギー反応を起こす患者もおり、治療をさらに複雑にする可能性があるため、投与量の調整や他の治療法への切り替えが必要となります。

エリグルスタット（セルデルガ）やミグルスタット（ザベスカ）のようなSRT治療薬は、経口投与が容易ではありますが、ミグルスタット（ザベスカ）は下痢、吐き気、体重減少などの重篤な消化器症状を伴うため、治療中止やコンプライアンス違反につながる可能性があり、エリグルスタット（セルデルガ）は忍容性は高い一方で、頭痛、疲労、めまいを引き起こすことがあるため、患者によっては十分な使用が制限されます。

ERTの最大の課題の1つは、終生（通常2週間ごと）の点滴静注を必要とすることです。このことは、患者とヘルスケアシステムの双方にいくつかのハードルをもたらします。患者は治療のために定期的にヘルスケア施設を訪れる必要があり、時間的な制約や旅費の増加につながります。特に遠隔地に住む患者にとっては困難です。継続的な通院や点滴は負担となり、患者のQOLを低下させます。発展途上諸国の患者にとって、専門クリニックや輸液センターへのアクセスは限られている場合があります。

全体として、現在のゴーシェ病治療、特にERTとSRTの副作用と長い治療期間は、治療市場の成長を妨げる重大な障壁です。これらの問題は患者のコンプライアンス不良、高い治療費、特に経済的・ヘルスケア的に制約のある地域での限定的な導入の一因となっています。さらに、治療が2型および3型ゴーシェ病の神経症状に対処していないという事実が、その有効性と訴求力をさらに制限しています。

これらの課題を克服するためには、治療選択肢の革新、副作用のより良い管理、治療法の手頃な価格と入手しやすさの改善が、市場成長の拡大に不可欠です。

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：疾患タイプ別
• スニペット：治療タイプ別
• スニペット：投与経路別
• スニペット：エンドユーザー別
• スニペット：地域別

第3章 市場力学

• 影響要因
  • 促進要因
    • 酵素補充療法および基質還元療法の拡大
  • 抑制要因
    • 副作用と長い治療期間
  • 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な企業
  • 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
• パイプライン分析
• 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
• 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 ゴーシェ病治療市場：疾病タイプ別

• 1型ゴーシェ病
• 2型ゴーシェ病
• 3型ゴーシェ病

第6章 ゴーシェ病治療市場：治療タイプ別

• 酵素補充療法（ERT）
  • イミグルセラーゼ（セレザイム）
  • ベラグルセラーゼアルファ（VPRIV）
  • タリグルセラーゼアルファ（エレリソ）
• 基質還元療法（SRT）
  • ミグルスタット（ザベスカ）
  • エリグルスタット（セルデルガ）
• 外科的治療
  • 骨髄移植
  • 関節置換手術
  • 脾臓摘出手術

第7章 ゴーシェ病治療市場：投与経路別

• 静脈内
• オーラル

第8章 ゴーシェ病治療市場：エンドユーザー別

• 病院
• 専門クリニック
• 学術研究機関
• 在宅ケア環境
• その他

第9章 ゴーシェ病治療市場：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
• Takeda Pharmaceuticals U.S.A., Inc.
• Pfizer Inc.
• Johnson & Johnson

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

The global Gaucher disease treatment market reached US$ 1.76 billion in 2024 and is expected to reach US$ 2.28 billion by 2033, growing at a CAGR of 2.9% during the forecast period 2025-2033.

Gaucher disease is a rare inherited metabolic disorder caused by a deficiency of an enzyme called glucocerebrosidase (also known as glucosylceramidase), which leads to the accumulation of a fatty substance called glucocerebroside in various organs of the body, primarily in the spleen, liver, bones and bone marrow. This accumulation can cause a wide range of symptoms that vary in severity depending on the type of Gaucher disease and the organs affected.

Gaucher disease treatment options such as enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) can significantly improve quality of life, but there is currently no cure. Gene therapy is emerging as a potential long-term solution, but it is still under development. The companies that are focusing on the ongoing research and treatment advancements in gene therapy are promising.

For instance, in July 2024, Spur Therapeutics announced that its gene therapy candidate for Gaucher disease would be moving to a Phase III trial after positive early data from a Phase I/II trial. The GALILEO-1 trial is investigating FLT201, an adeno-associated virus (AAV) gene therapy candidate, as a treatment for Gaucher disease. FLT-201 acts as a glucosylceramidase beta (GBA1) gene activator. Mutations in this gene cause Gaucher disease. The drug restores the function of the defective GBA1 gene and alleviates the condition.

Market Dynamics: Drivers & Restraints

Expansion of Enzyme Replacement and Substrate Reduction Therapies

The expansion of enzyme replacement and substrate reduction therapies is significantly driving the growth of the Gaucher disease treatment market and is expected to drive the market over the forecast period. Enzyme replacement therapy is the most common and established treatment for Type 1 Gaucher disease. It involves the intravenous administration of synthetic glucocerebrosidase, the enzyme deficient in individuals with Gaucher disease, which helps in breaking down glucocerebroside and preventing its accumulation in organs like the liver, spleen and bone marrow.

For instance, Imiglucerase (Cerezyme), the first ERT drug approved by the FDA, remains the gold standard, and other drugs like Velaglucerase alfa (Vpriv) and Taliglucerase alfa (Elelyso) are gaining traction in the market. These enzyme replacement therapies have demonstrated significant efficacy in treating organomegaly (enlarged spleen and liver) and bone-related complications in Gaucher patients.

Substrate reduction therapy (SRT) is an alternative or adjunct to enzyme replacement therapy, especially for patients who prefer oral medication over intravenous infusion or for those who cannot tolerate enzyme replacement therapy. SRT works by reducing the production of glucocerebroside, the substrate that accumulates in Gaucher disease. For instance, key drugs like Eliglustat (Cerdelga) and Miglustat (Zavesca) are the main substrate reduction therapies approved for Gaucher disease. These drugs can be taken orally, which increases patient compliance compared to the more invasive intravenous ERT.

Overall, the expansion of ERT and SRT has played a crucial role in the growth of the Gaucher disease treatment market. These therapies are not only improving patient outcomes but also enhancing treatment accessibility globally. The availability of oral therapies (SRT), in particular, offers significant convenience and helps increase patient adherence to treatment, while ERT continues to dominate due to its proven efficacy.

Side Effects & Long Treatment Duration

The side effects and long treatment durations associated with the treatment of Gaucher disease, particularly enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), present significant barriers that hamper the growth of the Gaucher disease treatment market. While these therapies are crucial for managing the disease, they come with certain challenges that may affect patient compliance, treatment adoption, and overall market expansion.

ERT, although effective, involves intravenous infusions of glucocerebrosidase (such as Cerezyme, Vpriv, and Elelyso), which are associated with several side effects that can impact patient adherence and overall treatment efficacy. Common side effects include fever, chills, headache, and nausea, often during or after the infusion. These reactions can cause discomfort and may discourage some patients from continuing treatment. Some patients develop allergic responses to the enzyme, which can further complicate treatment, leading to dose adjustments or switching to other forms of therapy.

SRT medications like Eliglustat (Cerdelga) and Miglustat (Zavesca), though easier to administer (oral), also come with significant side effects such as Miglustat (Zavesca) is associated with severe gastrointestinal symptoms such as diarrhea, nausea and weight loss, which can lead to discontinuation or non-compliance and Eliglustat (Cerdelga), while better tolerated, can cause headaches, fatigue and dizziness, limiting its full utilization in some patients.

One of the biggest challenges with ERT is that it requires lifelong intravenous infusions (typically every two weeks). This creates several hurdles for both patients and healthcare systems. Patients need to regularly visit healthcare facilities for treatment, leading to increased time commitments and travel expenses. This is especially difficult for patients living in remote areas. Continuous hospital visits and infusions can be burdensome and reduce the patient's quality of life. For patients in developing countries, access to specialized clinics and infusion centers may be limited.

Overall, the side effects and long treatment duration of current Gaucher disease treatments, particularly ERT and SRT, are significant barriers that hinder the growth of the treatment market. These issues contribute to poor patient compliance, high treatment costs, and limited adoption, especially in regions with financial or healthcare limitations. Additionally, the fact that treatments do not address the neurological symptoms of Type 2 and Type 3 Gaucher disease further limits their efficacy and appeal.

To overcome these challenges, innovation in treatment options, better management of side effects, and improvements in the affordability and accessibility of treatments will be essential for expanding market growth.

Segment Analysis

The global Gaucher disease treatment market is segmented based on disease type, treatment type, route of administration, end-user, and region.

Treatment Type:

The enzyme replacement therapy in the treatment type segment is expected to dominate with the highest market share.

Enzyme replacement therapy (ERT) has been the standard of care for Type 1 Gaucher disease for over two decades. It involves the intravenous infusion of a recombinant form of glucocerebrosidase (the enzyme deficient in Gaucher disease). ERT is highly effective in reducing spleen and liver enlargement, improving bone health, and increasing hemoglobin and platelet counts. For instance, Imiglucerase (Cerezyme), the first FDA-approved ERT, has been shown to significantly reduce splenomegaly and hepatomegaly, as well as improve bone mineral density and reduce painful bone crises.

Cerezyme (Imiglucerase) remains the market leader due to its long history, proven safety, and efficacy. It is used extensively worldwide and is widely accepted by physicians as the primary treatment for Type 1 Gaucher disease. In 2023, Cerezyme generated 720.02 million in net sales whereas, in 2024, Cerezyme generated USD 777.66 million, indicating its continued dominance in the treatment market. This dominance is due to the therapy's ability to address both the visceral (organ-related) and hematologic (blood-related) symptoms of the disease.

Additionally, Velaglucerase alfa (Vpriv) and Taliglucerase alfa (Elelyso), newer ERT products, also contribute to the growth of the ERT market, expanding treatment options for patients. These therapies are similar to Imiglucerase, offering additional options for patients who may not respond well to Cerezyme or who prefer a different treatment formulation.

One of the reasons ERT continues to dominate is that it is the primary long-term solution for Type 1 Gaucher disease. Patients often need lifelong treatment to manage their disease, which leads to continued use of ERT over extended periods. While the treatment requires intravenous infusions every two weeks, the efficacy in controlling symptoms makes it a preferred choice for many patients and healthcare providers.

Geographical Analysis

The North America region is expected to hold a significant position in the Gaucher disease treatment market with the highest market share

Gaucher disease is more commonly diagnosed in North America, especially in the United States, compared to other regions due to a higher level of awareness, better healthcare systems, and more frequent genetic screening. According to the National Institute of Health (NIH), among the Ashkenazi Jewish population, Gaucher disease is the most common genetic disorder, with an incidence of approximately 1 in 450 persons. In the general public, Gaucher disease affects approximately 1 in 100,000 persons. As a result, North America has a higher number of diagnosed cases compared to other regions.

North America boasts a highly developed healthcare system, providing patients with access to state-of-the-art treatments such as enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). The availability of specialized treatment centers, including academic medical centers and rare disease clinics, ensures that patients with Gaucher disease receive the highest level of care.

For instance, the U.S. Food and Drug Administration (FDA) has been instrumental in the approval of key Gaucher disease treatments, such as Cerezyme (Imiglucerase), Vpriv (Velaglucerase alfa), and Elelyso (Taliglucerase alfa). The rapid approval process and support for biopharmaceutical innovation have ensured that the latest therapies are accessible to patients in North America soon after they are developed.

North America is a global leader in pharmaceutical research and development, with many leading pharmaceutical companies based in the U.S. actively involved in clinical trials and the development of innovative therapies for Gaucher disease. Companies such as Genzyme (Sanofi), Pfizer, Freeline Therapeutics, and Shire (part of Takeda) play a significant role in driving innovation and improving treatment options by performing clinical trials for Gaucher disease patients.

For instance, in May 2024, Freeline Therapeutics announced new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, showing substantial reductions in glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response, in patients with persistently high levels despite years of treatment with currently approved therapies, as well as early signs of clinical improvements in bone marrow burden and fatigue. FLT201 continues to demonstrate a favorable safety and tolerability profile.

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 Gaucher disease treatment market report delivers a detailed analysis with 52 key tables, more than 57 visually impactful figures, and 179 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 Treatment Type
• 2.5. Snippet by Route of Administration
• 2.6. Snippet by End-User
• 2.7. Snippet by Region

3. Dynamics

• 3.1. Impacting Factors
  • 3.1.1. Drivers
    • 3.1.1.1. Expansion of Enzyme Replacement and Substrate Reduction Therapies
  • 3.1.2. Restraints
    • 3.1.2.1. Side Effects & Long Treatment Duration
  • 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. Pipeline Analysis
• 4.6. Regulatory and Reimbursement Landscape
  • 4.6.1. North America
  • 4.6.2. Europe
  • 4.6.3. Asia Pacific
  • 4.6.4. Latin America
  • 4.6.5. Middle East & Africa
• 4.7. Porter's Five Forces Analysis
• 4.8. Supply Chain Analysis
• 4.9. Patent Analysis
• 4.10. SWOT Analysis
• 4.11. Unmet Needs and Gaps
• 4.12. Recommended Strategies for Market Entry and Expansion
• 4.13. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
• 4.14. Pricing Analysis and Price Dynamics
• 4.15. Key Opinion Leaders

5. Gaucher 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. Type 1 Gaucher Disease*
  • 5.2.1. Introduction
  • 5.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 5.3. Type 2 Gaucher Disease
• 5.4. Type 3 Gaucher Disease

6. Gaucher Disease Treatment Market, By Treatment Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment Type
  • 6.1.2. Market Attractiveness Index, By Treatment Type
• 6.2. Enzyme Replacement Therapy (ERT)*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 6.2.3. Imiglucerase (Cerezyme)
  • 6.2.4. Velaglucerase alfa (VPRIV)
  • 6.2.5. Taliglucerase alfa (Elelyso)
• 6.3. Substrate Reduction Therapy (SRT)
  • 6.3.1. Miglustat (Zavesca)
  • 6.3.2. Eliglustat (Cerdelga)
• 6.4. Surgical Treatment
  • 6.4.1. Bone Marrow Transplant
  • 6.4.2. Joint Replacement Surgery
  • 6.4.3. Spleen Removal Surgery

7. Gaucher 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. Intravenous*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Oral

8. Gaucher Disease Treatment Market, By End-User

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 8.1.2. Market Attractiveness Index, By End-User
• 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. Academic and Research Institutes
• 8.5. Homecare Settings
• 8.6. Others

9. Gaucher 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 Treatment Type
  • 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 End-User
  • 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 Treatment Type
  • 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 End-User
  • 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 Treatment Type
  • 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 End-User
  • 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 Treatment Type
  • 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 End-User
  • 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 Treatment Type
  • 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 End-User

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. Takeda Pharmaceuticals U.S.A., Inc.
• 11.3. Pfizer Inc.
• 11.4. Johnson & Johnson

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