表紙:ファブリー病治療の世界市場(2021年~2028年)
市場調査レポート
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974574

ファブリー病治療の世界市場(2021年~2028年)

Global Fabry Disease Treatment Market - 2021-2028

出版日: | 発行: DataM Intelligence | ページ情報: 英文 180 Pages | 納期: 約2営業日

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ファブリー病治療の世界市場(2021年~2028年)
出版日: 2021年07月09日
発行: DataM Intelligence
ページ情報: 英文 180 Pages
納期: 約2営業日
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  • 概要
  • 目次
概要

世界のファブリー病治療薬市場の医薬品市場の成長は、ファブリー病患者数の増加、シャペロン治療などの新しい治療法の採用増加によって推進されており、市場成長は加速しています。これに加え、広範な研究開発活動と基質減少療法や酵素補充療法を含む有望なパイプライン製品の潜在的な承認が、予測期間にわたって成長を促進すると予測されています。

また、ファブリー病の治療に適応される強力な投薬パイプラインの存在も、予測期間中の世界のファブリー病治療市場の成長を促進する主要な要因であると予想されます。最近では、主要地域のさまざまな規制機関がファブリー病の治療のための新しい薬を承認しています。各地域における新しい薬の承認と発売は、予測期間にわたって世界市場の成長を大幅に後押しすると期待されています。

当レポートは、世界のファブリー病治療市場について調査しており、市場の機会や影響、成長および抑制要因、薬剤・治療・投与経路・流通チャネル・地域別の市場分析、競合情勢、主要企業のプロファイルなどの情報を提供しています。

目次

第1章 調査手法と範囲

  • 調査手法
  • 調査目的と調査範囲

第2章 市場の定義と概要

第3章 エグゼクティブサマリー

  • 医薬品別
  • 治療別
  • 投与経路別
  • 流通チャネル別
  • 地域別

第4章 市場力学

  • 市場に影響を与える要因
    • 成長要因
    • 抑制要因
    • 市場機会
    • 影響分析

第5章 業界分析

  • 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
  • 償還分析
  • アンメットニーズ

第6章 COVID-19の分析

  • 市場におけるCovid-19の分析
  • Covid-19における価格の力学
  • 需要と供給のスペクトル
  • パンデミック時の市場に関連する政府のイニシアチブ
  • メーカーの戦略的イニシアチブ
  • 結論

第7章 市場分析:薬剤別

  • イントロダクション
  • 市場規模分析および前年比成長分析
  • 市場魅力指数
    • 承認された薬
    • パイプライン薬

第8章 市場分析:治療別

  • イントロダクション
  • 市場規模分析および前年比成長分析
  • 市場魅力指数
    • 酵素補充療法(ERT)
    • シャペロン治療
    • 基質減少療法(SRT)
    • その他

第9章 市場分析:投与経路別

  • イントロダクション
  • 市場規模分析および前年比成長分析
  • 市場魅力指数
    • 経口
    • 静脈内

第10章 市場分析:流通チャネル別

  • イントロダクション
  • 市場規模分析および前年比成長分析
  • 市場魅力指数
    • 病院薬局
    • 小売薬局
    • オンライン薬局
    • その他

第11章 市場分析:地域別

  • イントロダクション
  • 市場規模分析、および前年比成長分析
  • 市場魅力指数
  • 北米
  • 欧州
  • 南米
  • アジア太平洋
  • 中東とアフリカ

第12章 競合情勢

  • 競合シナリオ
  • 市場の位置づけ/シェア分析
  • 合併と買収の分析

第13章 企業プロファイル

  • Amgen Inc.
    • 企業概要
    • 製品ポートフォリオ・概要
    • 要点
    • 財務概要
  • Amicus Therapeutics, Inc.
  • Bristol-Myers Squibb Company
  • GlaxoSmithKline
  • iBio, Inc.
  • Neuraltus Pharmaceuticals, Inc.
  • Novartis AG
  • Pfizer Inc.
  • AVROBIO, Inc.,
  • Idorsia Pharmaceuticals Ltd

第14章 重要考察

第15章 DataM

  • 付録
  • 当社・サービスについて
  • お問い合わせ
目次
Product Code: DMPH2867

Market Overview

The global Fabry Disease Treatment Market size was valued US$ XX billion in 2020 and is estimated to reach US$ XX billion by 2028, growing at a CAGR of XX % during the forecast period (2021-2028).

Fabry disease also called as Anderson-Fabry disease and alpha-galactosidase-A deficiency is an X-linked, hereditary, lysosomal storage disease caused due to paucity of an enzyme α galactosidase A (an enzyme required to metabolize lipids, fat-like substances that include oils, waxes, and fatty acids).

Market Dynamics

The global fabry disease treatment market growth is driven by the expanding base of patients suffering from fabry disease coupled with increased adoption of novel therapies such as chaperone treatment has helped the market gain momentum. Besides this, extensive R&D activities and potential approval of promising pipeline products including substrate reduction therapies and enzyme replacement therapies are projected to drive the growth of the over the forecast period.

The presence of robust medication pipeline indicated for treatment of fabry disease is expected to drive the growth in the forecast period

The presence of robust medication pipeline indicated for treatment of fabry disease is also expected to be a major factor fueling growth of global fabry disease treatment market over the forecast period.

In recent past, various regulatory bodies of key regions have approved novel medications for treatment of fabry disease. Approval and launch of such novel medications is expected to significantly support global fabry disease treatment market growth over the forecast period.

For instance, in August 2018, Amicus Therapeutics, Inc., received the U.S. Food and Drug Administration (FDA) approval for its drug Galafold (migalastat). It is the first oral medication indicated for the treatment of adults with Fabry disease.

Furthermore, key players in the market are majorly investing in development of novel therapies for Fabry disease treatment, such strategic funding's by various players is expected to drastically fuel global fabry disease treatment market growth over the forecast period.

For instance, in February 2018, AvroBio Inc., closed US$ 60 million financing to support the phase 2 study of its novel gene therapy, AVR-RD-01 underdevelopment for the treatment of Fabry disease. Considering these factors will drive the growth of the market in the forecast period.

Lack of a curative solution for Fabry disease treatment are projected, expected to drive the market growth

Patients suffering from Fabry disease commonly experience stroke, extreme pain, early heart disorder, compromised kidney function often leading to full kidney failure, and disabling gastrointestinal symptoms. Due to this lack of a curative solution for Fabry disease treatment options aims to manage complications associated with disease progression and giving symptomatic relief. Many of times, various patients remain undiagnosed due to moderate symptoms. Considering these factors, the global fabry disease market is projected to grow at a significate rate in the forecast period.

Low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals is likely to hinder the market growth

However, fabry disease is a very rare disease affecting around 1 individual in 50,000 (according to the National Fabry Disease Foundation), the diagnosis of Fabry disease is confirmed by demonstrating an enzyme deficiency in males and by identifying the specific GLA gene mutation in males and females. Hence, low diagnostic rate due to unavailability of such diagnostic procedures and trained professionals in emerging economies are expected to be major factor restraining global fabry disease treatment market growth.

COVID-19 Impact Analysis

Coronavirus Disease-19 (COVID-19), caused by the SARS-CoV-2 virus was labeled as a global pandemic and patients with fabry disease can be considered at high risk of developing severe complications. The impact of COVID-19 on patients with fabry disease receiving enzyme replacement therapy is still unknown. Many patients who receive treatment in the hospital experienced infusion disruptions due to fear of infection. Effects of temporary treatment interruption was described in more detail in other lysosomal storage diseases, but the recommencement of therapy does not fully reverse clinical decline due to the temporary discontinuation. When possible, home-therapy seems to be the most efficient way to maintain enzyme replacement therapy access during pandemic. These factors will drive the growth of the market in the forecast period.

Segment Analysis

Enzyme replacement therapy, segment is expected to grow at the fastest CAGR during the forecast period (2021-2028)

The standard of care for disease management is enzyme replacement therapy. Although, both the enzyme replacement therapy, Sanofi's Fabrazyme and Shire's Replagal, have been approved in Europe, only Fabrazyme has been approved in the U.S. The focus of current clinical trials is to improve safety and efficacy profile of enzyme replacement therapy and introduction of novel oral therapies that can eliminate the need for intravenous infusions. Recently Amicus Therapeutics' Galafold has been approved as the first oral chaperone therapy for the treatment of adult patients in the U.S., Canada, EU, Japan, Australia, Israel, and South Korea.

Furthermore, Enzyme Replacement Therapy is an infusion (injection) of an enzyme designed to substitute for the missing ARSA enzyme that causes metachromatic leukodystrophy. ERT has been successfully used for fabry disease treatment. These factors are driving the growth of the market in the forecast period.

Pipeline Analysis

Takeda Pharmaceutical Company is developing the MLD ERT therapy, which they refer to as SHP-611. The initial development and clinical trial work were performed by Shire PLC, which was acquired by Takeda in early 2019. SHP-611 is based on several decades of Shire ERT research, expertise, and commercialization for other lysosomal disease products.

Registration - MLD ERT is still in Phase I/II clinical trial and has not been submitted for any agency review or registrations. As of Q3'2019, Takeda is forecasting regulatory approval and commercialization in 2023 based on the current clinical trial.

2012 Clinical Trial - A phase I/II dose-escalation study of 24 patients receiving HGT-1110 (Shire's internal product reference code) with infusions every other week started in February 2012 and completed in January 2017. The trial analysis provided guidance to increase the dose frequency and amount for the current trial.

Orphan Designation - SHP-611 has been granted Orphan Designation by the EMA for Europe (EU/3/10/813)

Phase II ERT Trial Recruiting Status and Sites

Takeda opened recruiting for their Phase II Clinical Trial for SHP611, an ERT therapy for MLD in April 2019. Their marketing name for the study is the EMBOLDEN Study. The trial will include pre-symptomatic, and for the first time, symptomatic late infantiles (and their younger pre-symptomatic siblings). For participation with symptomatic children.

They have announced 24 sites, including for the first time, 10 sites in the US. The sites will open one by one throughout 2019 and 2020.

infections which leads to a condition called immune reconstitution inflammatory syndrome (IRIS).

Geographical Analysis

North America region holds the largest market share global Fabry Disease Treatment Market

North America region is dominating the global Fabry Disease Treatment Market accounted for the largest market share in 2020. Higher adoption of novel therapies, better healthcare facilities, and favorable reimbursement policies are playing a vital role in the growth of the market in the region. Coverage of expensive medications such as Fabrazyme by health insurance programs and favorable governmental healthcare policies are also prompting drug companies to increase R&D investments in the field of rare diseases.

Fabry Disease Treatment Market Competitive Landscape

The Fabry Disease Treatment Market is moderately competitive with presence of local as well as global companies. Some of the key players which are contributing to the growth of the market include Amgen Inc., Amicus Therapeutics, Inc., Bristol-Myers Squibb Company, GlaxoSmithKline, iBio, Inc., Neuraltus Pharmaceuticals, Inc., Novartis AG, Pfizer Inc., AVROBIO, Inc., Idorsia Pharmaceuticals Ltd, Protalix, Sanofi, Shire, Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd among others. The major players are adopting several growth strategies such as product launches, acquisitions, and collaborations, which are contributing to the growth of the Fabry Disease Treatment Market globally.

Sanofi S.A

Overview: Sanofi is a healthcare company, focused on patient needs and engaged in the research, development, manufacture and marketing of therapeutic solutions. Its segments are Pharmaceuticals, Human Vaccines (Vaccines), and Other. The Pharmaceuticals segment comprises the commercial operations of various franchises, including Specialty Care (Rare Diseases, Multiple Sclerosis, and Oncology), Diabetes and Cardiovascular, Established Prescription Products, Consumer Healthcare and Generics, and research and development, production and marketing activities for all of the Company's pharmaceuticals operations.

Product Portfolio: The Company's portfolio comprised of drugs, generic medicines, food supplement, and cosmetics.

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  • Visualize the composition of the fabry disease treatment market segmentation by drugs, treatment, route of administration, distribution channel and region highlighting the key commercial assets and players.
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  • Excel data sheet with thousands of data points of fabry disease treatment Market - level 4/5 segmentation.
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  • Product mapping in excel for the key product of all major market players

The global fabry disease treatment market report would provide an access to an approx. 69 market data table, 66 figures and 300 pages.

Target Audience

  • Service Providers/ Buyers
  • Industry Investors/Investment Bankers
  • Education & Research Institutes
  • Research Professionals
  • Emerging Companies
  • Manufacturers

Market Segmentation

Global Fabry Disease Treatment Market - By Drugs

  • Approved Drugs

Agalsidase Beta (Fabrazyme/Replagal)

Migalastat (Galafold)

  • Pipeline Drugs

Global Fabry Disease Treatment Market - By Treatment

  • Enzyme Replacement Therapy (ERT)
  • Chaperone Treatment
  • Substrate Reduction Therapy (SRT)
  • Others

Global Fabry Disease Treatment Market - By Route of Administration

  • Oral
  • Intravenous

Global Fabry Disease Treatment Market - By Distribution Channel

  • Hospitals Pharmacies
  • Retail Pharmacies
  • Online pharmacies
  • Others

Global Fabry Disease Treatment Market - By Region

  • North America
  • South America
  • Europe
  • Asia
  • Middle East & Africa

Table of Contents

1. Global Fabry Disease Treatment Market Methodology and Scope

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

2. Global Fabry Disease Treatment Market - Market Definition and Overview

3. Global Fabry Disease Treatment Market - Executive Summary

  • 3.1. Market Snippet by Drugs
  • 3.2. Market snippet by Treatment
  • 3.3. Market Snippet by Route of Administration
  • 3.4. Market Snippet by Distribution Channel
  • 3.5. Market Snippet by Region

4. Global Fabry Disease Treatment Market - Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Lack of a curative solution for Fabry disease treatment are projected
      • 4.1.1.2. The presence of robust medication pipeline indicated for treatment
    • 4.1.2. Restraints:
      • 4.1.2.1. Low diagnostic rate due to unavailability of such diagnostic procedures
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Global Fabry Disease Treatment Market - Industry Analysis

  • 5.1. Porter's Five Forces Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Pricing Analysis
  • 5.4. Regulatory Analysis
  • 5.5. Reimbursement Analysis
  • 5.6. Unmet Needs

6. Global Fabry Disease Treatment Market - COVID-19 Analysis

  • 6.1. Analysis of Covid-19 on the Market
    • 6.1.1. Before COVID-19 Market Scenario
    • 6.1.2. Present COVID-19 Market Scenario
    • 6.1.3. After COVID-19 or Future Scenario
  • 6.2. Pricing Dynamics Amid Covid-19
  • 6.3. Demand-Supply Spectrum
  • 6.4. Government Initiatives Related to the Market During Pandemic
  • 6.5. Manufacturers Strategic Initiatives
  • 6.6. Conclusion

7. Global Fabry Disease Treatment Market - By Drugs

  • 7.1. Introduction
    • 7.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Drugs Segment
    • 7.1.2. Market Attractiveness Index, By Drugs Segment
  • 7.2. Approved Drugs *
    • 7.2.1. Introduction
    • 7.2.2. Market Size Analysis, US$ Million, 2018-2028 and Y-o-Y Growth Analysis (%), 2020-2028
    • 7.2.3. Agalsidase Beta (Fabrazyme/Replagal)
    • 7.2.4. Migalastat (Galafold)
  • 7.3. Pipeline Drugs

8. Global Fabry Disease Treatment Market - By Treatment

  • 8.1. Introduction
    • 8.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 8.1.2. Market Attractiveness Index, By Treatment
  • 8.2. Enzyme Replacement Therapy (ERT) *
    • 8.2.1. Introduction
    • 8.2.2. Market Size Analysis, US$ Million, 2018-2028 and Y-o-Y Growth Analysis (%), 2020-2028
  • 8.3. Chaperone Treatment
  • 8.4. Substrate Reduction Therapy (SRT)
  • 8.5. Others

9. Global Fabry Disease Treatment Market - By Route of Administration

  • 9.1. Introduction
    • 9.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration- Segment
    • 9.1.2. Market Attractiveness Index, By Route of Administration- Segment
  • 9.2. Oral *
    • 9.2.1. Introduction
      • 9.2.1.1. Market Size Analysis, US$ Million, 2018-2028 and Y-o-Y Growth Analysis (%), 2020-2028
    • 9.2.2. Intravenous

10. 9. Global Fabry Disease Treatment Market - By Distribution Channel

  • 10.1. Introduction
    • 10.1.1. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel- Segment
    • 10.1.2. Market Attractiveness Index, By Distribution Channel- Segment
  • 10.2. Hospitals Pharmacies*
    • 10.2.1. Introduction
    • 10.2.2. Market Size Analysis, US$ Million, 2018-2028 and Y-o-Y Growth Analysis (%), 2020-2028
  • 10.3. Retail Pharmacies
  • 10.4. Online pharmacies
  • 10.5. Others

11. Global Fabry Disease Treatment Market - By Region

  • 11.1. Introduction
    • 11.1.1. Market Size Analysis, US$ Million, 2018-2028 and Y-o-Y Growth Analysis (%), 2020-2028, By Region
    • 11.1.2. Market Attractiveness Index, By Region
  • 11.2. North America
    • 11.2.1. Introduction
    • 11.2.2. Key Region-Specific Dynamics
    • 11.2.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Drugs
    • 11.2.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 11.2.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.2.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.2.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.2.7.1. U.S.
      • 11.2.7.2. Canada
      • 11.2.7.3. Mexico
  • 11.3. Europe
    • 11.3.1. Introduction
    • 11.3.2. Key Region-Specific Dynamics
    • 11.3.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Drugs
    • 11.3.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 11.3.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.3.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.3.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.3.7.1. Germany
      • 11.3.7.2. U.K.
      • 11.3.7.3. France
      • 11.3.7.4. Italy
      • 11.3.7.5. Spain
      • 11.3.7.6. Rest of Europe
  • 11.4. South America
    • 11.4.1. Introduction
    • 11.4.2. Key Region-Specific Dynamics
    • 11.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Drugs
    • 11.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 11.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.4.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.4.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.4.7.1. Brazil
      • 11.4.7.2. Argentina
      • 11.4.7.3. Rest of South America
  • 11.5. Asia Pacific
    • 11.5.1. Introduction
    • 11.5.2. Key Region-Specific Dynamics
    • 11.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Type
    • 11.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 11.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.5.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel
    • 11.5.7. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 11.5.7.1. China
      • 11.5.7.2. India
      • 11.5.7.3. Japan
      • 11.5.7.4. Australia
      • 11.5.7.5. Rest of Asia Pacific
  • 11.6. Middle East and Africa
    • 11.6.1. Introduction
    • 11.6.2. Key Region-Specific Dynamics
    • 11.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Drugs
    • 11.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment
    • 11.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Route of Administration
    • 11.6.6. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Distribution Channel

12. Global Fabry Disease Treatment Market - Competitive Landscape

  • 12.1. Key Developments and Strategies
  • 12.2. Company Share Analysis
  • 12.3. Product Benchmarking
  • 12.4. List of key companies to watch
  • 12.5. List of the company with disruptive technology
  • 12.6. List of start-up companies

13. Global Fabry Disease Treatment Market- Company Profiles

  • 13.1. Amgen Inc.*
    • 13.1.1. Company Overview
    • 13.1.2. Product Portfolio and Description
    • 13.1.3. Key Highlights
    • 13.1.4. Financial Overview
  • 13.2. Amicus Therapeutics, Inc.
  • 13.3. Bristol-Myers Squibb Company
  • 13.4. GlaxoSmithKline
  • 13.5. iBio, Inc.
  • 13.6. Neuraltus Pharmaceuticals, Inc.
  • 13.7. Novartis AG
  • 13.8. Pfizer Inc.
  • 13.9. AVROBIO, Inc.,
  • 13.10. Idorsia Pharmaceuticals Ltd

LIST NOT EXHAUSTIVE

14. Global Fabry Disease Treatment Market - DataM

  • 14.1. Appendix
  • 14.2. About Us and Services
  • 14.3. Contact Us