Global Fabry Disease Treatment Market - 2023-2030
The Global Fabry Disease Treatment Market size reached US$ 1,876.7 million in 2022 and is projected to witness lucrative growth by reaching up to US$ 3,287.2 million by 2030. The global Fabry disease treatment market is expected to exhibit a CAGR of 7.4% during the forecast period (2023-2030). The key market trend is the increasing focus on precision medicine and personalized therapies.
For instance, Personalized medicine, therapies tailored to patients' genetic and molecular makeup, is expected to improve outcomes and help direct patients away from therapies that don't work for them. It is aligned with the Triple Aim of improving patient experience, population health, and cost reduction.
Fabry disease is a rare genetic disorder characterized by the deficiency or malfunction of an enzyme called alpha-galactosidase A (AGA). This enzyme is responsible for breaking down a fatty substance called globotriaosylceramide (GL-3) or globotriaosylsphingosine (Lyso-GL-3). The accumulation of GL-3 or Lyso-GL-3 in various tissues and organs can lead to severe symptoms and complications.
The Fabry disease treatment market is driven by factors such as increasing awareness and diagnosis, increasing demand for novel therapies such as chaperone therapies and enzyme replacement therapies, advancements in treatment options, increasing R&D activities, and technological advancements.
For instance, in March 2023, Chiesi Global Rare Diseases, a part of the Chiesi Group and Protalix BioTherapeutics, a biopharmaceutical company announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorization for PRX-102. The therapy also known as pegunigalsidase alfa, is the first and only pegylated enzyme for the treatment of adult patients with Fabry disease
Market Dynamics
Increasing Demand For Novel Therapies
The increasing demand for novel therapies is indeed a major factor driving the market share in the Fabry disease treatment market. There is a growing need for more effective and innovative treatment options that can provide better outcomes for patients with Fabry disease. The limitations and challenges associated with current therapies, such as enzyme replacement therapy (ERT), have created a demand for alternative approaches.
The development of novel therapies, such as chaperone therapies and substrate reduction therapies, has generated significant interest and excitement in the field. These therapies offer potential advantages such as improved efficacy, convenience, and reduced treatment burden compared to traditional ERT. They provide additional treatment options for patients who may not respond optimally to ERT or have limitations in accessing or receiving ERT.
High Cost of the Treatment and Limited Treatment Options
The high cost of treatment and limited treatment options can present challenges and potentially hamper the growth of the Fabry disease treatment market. These factors can impact both patients and healthcare systems.
Fabry disease treatments, such as enzyme replacement therapy (ERT) and other emerging therapies, can be costly. The high cost of these treatments can create financial barriers for patients, particularly in regions with limited healthcare coverage or inadequate reimbursement policies. The affordability of therapies can significantly influence access to treatment and market penetration.
While there have been advancements in Fabry disease treatment, the availability of treatment options remains limited compared to more prevalent conditions. This limited choice can restrict patient access to therapies that are most suitable for their specific disease profile or treatment preferences. It can also limit competition in the market, potentially affecting affordability and innovation.
COVID-19 Impact Analysis
The COVID-19 pandemic has had a profound impact on the healthcare industry, including the Fabry disease treatment market. The economic consequences of the pandemic have affected healthcare systems, payers, and patients. Healthcare budgets may have been strained, leading to potential challenges in funding expensive treatments like ERT.
Patients may have faced financial hardships due to job loss, reduced incomes, or changes in insurance coverage, making it difficult to afford Fabry disease treatments or access necessary healthcare services.
The pandemic had an impact on ongoing research activities and clinical trials. Many trials were temporarily halted or experienced delays due to restrictions on patient recruitment, site closures, and prioritization of COVID-19 research. This may have slowed down the development and evaluation of new treatments or limited access to investigational therapies for Fabry disease patients.
Russia-Ukraine Conflict Analysis
The Russia-Ukraine conflict has had a significant impact on the Fabry disease treatment market. The disruptions caused by a conflict could affect the production, distribution, and availability of medications, including enzyme replacement therapy (ERT). It may also disrupt the delivery of healthcare services, such as access to specialized clinics, diagnostic facilities, or healthcare professionals.
The conflict may disrupt research and development activities related to Fabry disease treatments. Clinical trials, collaborations, and research projects involving institutions or companies in the affected regions may face delays or interruptions. This can slow down the development of new therapies or the evaluation of existing treatments, affecting the overall advancement of the Fabry disease treatment market.
Segment Analysis
The global Fabry disease treatment market is segmented based on drugs, treatment, route of administration, distribution channel, and region.
Based on Treatment, the Enzyme Replacement Therapy (ERT) Segment is Expected to Dominate the Market Share
The market for enzyme replacement therapy (ERT) holds the 65.2% of the global Fabry disease treatment market share in 2022. Currently, there are two approved ERT products for Fabry disease: agalsidase alfa (Replagal) and agalsidase beta (Fabrazyme). These products have been in the market for several years and have established a significant market presence. The availability of approved ERT options provides healthcare professionals and patients with a reliable and recognized treatment approach.
In May 2023, Protalix BioTherapeutics, a biopharmaceutical company, and Chiesi Group, a specialty pharmaceutical company developed a drug called Elfabrio is approved by FDA for treating Fabry disease, a rare inherited metabolic disorder. The drug, Elfabrio, is an enzyme replacement therapy.
Geographical Analysis
North America Holds the Largest Market Share Due to the Increasing Adoption of Novel Therapies and the Presence of a Strong Healthcare Infrastructure
North America holds the 43.2% market share for the Fabry disease treatment market in 2022 due to the increasing adoption of novel therapies and the presence of strong healthcare infrastructure in the region.
North America has been at the forefront of adopting and integrating novel therapies for Fabry disease treatment. The region has a robust research and development ecosystem, which facilitates the discovery and development of innovative treatment options. The introduction of alternative therapies, such as chaperone therapies and substrate reduction therapies, has gained traction in North America, providing additional treatment choices for patients.
Competitive Landscape
The major global players in the market include Sanofi S.A, Takeda Pharmaceutical Company Ltd, Teva Pharmaceutical Industries Ltd., Amicus Therapeutics Inc., Amgen Inc., Bristol-Myers Squibb Company, Neuraltus Pharmaceuticals Inc., Novartis AG, Pfizer Inc., and Idorsia Pharmaceuticals Ltd.
Why Purchase the Report?
• To visualize the global Fabry disease treatment market segmentation based on drugs, treatment, route of administration, 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 Fabry 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 Fabry disease treatment market report would provide approximately 54 tables, 46 figures, and 195 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies
- 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 Drugs
- 3.2. Snippet by Treatment
- 3.3. Snippet by Route of Administration
- 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 Demand for Novel Therapies
- 4.1.1.2. Increasing R&D Activities
- 4.1.2. Restraints
- 4.1.2.1. High Cost of the Treatment
- 4.1.2.2. Limited treatment options
- 4.1.3. Opportunity
- 4.1.3.1. Advancements in Treatment Options
- 4.1.4. Impact Analysis
- 5. Industry Analysis
- 5.1. Porter's 5 Forces Analysis
- 5.2. Supply Chain Analysis
- 5.3. Pricing Analysis
- 5.4. Regulatory Analysis
- 6. COVID-19 Analysis
- 6.1. Analysis of COVID-19
- 6.1.1. Scenario Before COVID-19
- 6.1.2. Scenario During COVID-19
- 6.1.3. Scenario Post COVID-19
- 6.2. Pricing Dynamics Amid COVID-19
- 6.3. Demand-Supply Spectrum
- 6.4. Government Initiatives Related to the Market During the Pandemic
- 6.5. Manufacturer’s Strategic Initiatives
- 6.6. Conclusion
- 7. By Drugs
- 7.1. Introduction
- 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drugs
- 7.1.2. Market Attractiveness Index, By Drugs
- 7.2. Agalsidase Beta (Fabrazyme/Replagal)*
- 7.2.1. Introduction
- 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 7.3. Migalastat (Galafold)
- 7.4. Others
- 8. 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 and Y-o-Y Growth Analysis (%)
- 8.3. Chaperone Treatment
- 8.4. Substrate Reduction Therapy (SRT)
- 8.5. Others
- 9. By Route of Administration
- 9.1. Introduction
- 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
- 9.1.2. Market Attractiveness Index, By Route of Administration
- 9.2. Oral*
- 9.2.1. Introduction
- 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 9.3. Parenteral
- 9.4. Others
- 10. By Distribution Channel
- 10.1. Introduction
- 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Distribution Channel
- 10.1.2. Market Attractiveness Index, By Distribution Channel
- 10.2. Hospital Pharmacies*
- 10.2.1. Introduction
- 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 10.3. Retail Pharmacies
- 10.4. Online Pharmacies
- 10.5. Others
- 11. By Region
- 11.1. Introduction
- 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), 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. The 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. The UK
- 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 Drugs
- 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. Competitive Landscape
- 12.1. Competitive Scenario
- 12.2. Market Positioning/Share Analysis
- 12.3. Mergers and Acquisitions Analysis
- 13. Company Profiles
- 13.1. Sanofi S.A.*
- 13.1.1. Company Overview
- 13.1.2. Product Portfolio and Description
- 13.1.3. Financial Overview
- 13.1.4. Key Developments
- 13.2. Takeda Pharmaceutical Company Ltd
- 13.3. Teva Pharmaceutical Industries Ltd.
- 13.4. Amicus Therapeutics Inc.
- 13.5. Amgen Inc.
- 13.6. Bristol-Myers Squibb Company
- 13.7. Neuraltus Pharmaceuticals Inc.
- 13.8. Novartis AG
- 13.9. Pfizer Inc.
- 13.10. Idorsia Pharmaceuticals Ltd.
- LIST NOT EXHAUSTIVE
- 14. Appendix
- 14.1. About Us and Services
- 14.2. Contact Us
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