This Fabry Disease Treatment market report provides a comprehensive analysis of the market’s characteristics, size, and growth, including segmentation, regional and country-level breakdowns, competitive landscape, market shares, trends, and strategies. It also tracks historical and forecasted market growth across various geographies.
The fabry disease treatment market size has grown strongly in recent years. It will grow from $2.09 billion in 2024 to $2.28 billion in 2025 at a compound annual growth rate (CAGR) of 9.1%. The growth in the historic period can be attributed to increasing awareness and diagnosis rates, increasing adoption of enzyme replacement therapies, increasing use of chaperone therapies, increasing healthcare infrastructure, rise in investment in rare disease research, increasing population, and increasing regulatory incentives for orphan drugs.
The fabry disease treatment market size is expected to see strong growth in the next few years. It will grow to $3.2 billion in 2029 at a compound annual growth rate (CAGR) of 8.8%. The growth in the forecast period can be attributed to increasing prevalence of the disease, rising awareness and diagnosis rates, expanding healthcare infrastructure, growing investment in rare disease research, growing patient awareness, and expanding reimbursement policies. Major trends in the forecast period include advancements in gene therapy, advancements in enzyme replacement therapies, advancements in diagnostic techniques, advancements in novel therapies, and integration of digital health technologies for patient management.
The growth of the Fabry disease treatment market is expected to be driven by the increasing prevalence of renal diseases. Renal diseases, or kidney diseases, encompass conditions that affect kidney function and are on the rise due to genetic factors, lifestyle choices, environmental influences, and underlying health issues. Fabry disease, specifically caused by the buildup of globotriaosylceramide (Gb3) in kidney cells, results in kidney damage and dysfunction. This emphasizes the urgent need for early detection and intervention to prevent kidney-related complications. For instance, recent data from the Australian Bureau of Statistics revealed a 0.2% increase in kidney disease cases over the past decade, with 246,200 people (1% of the population) affected in 2022, highlighting the escalating prevalence of renal diseases and their impact on the fabry disease treatment market.
Key players in the Fabry disease treatment market are actively developing plant cell-based therapies to enhance treatment efficacy, reduce costs, and improve patient outcomes through innovative and sustainable methods. Plant cell-based treatments for fabry disease involve using plant cells to produce deficient enzymes found in individuals with fabry disease. These plant-derived enzymes serve as a therapeutic option to replace the missing or defective enzymes in the body, aiming to alleviate symptoms and enhance patients' overall health. For example, ELFABRIO, an enzyme replacement therapy developed by Chiesi Global Rare Diseases and Protalix BioTherapeutics Inc., was approved in May 2023. ELFABRIO utilizes recombinant human α-Galactosidase-A enzyme produced in plant-cell culture and modified with PEGylation to extend its half-life significantly. This innovative treatment approach reduces treatment frequency, increases convenience, and improves symptom management, ultimately enhancing the quality of life for fabry disease patients.
In March 2024, CENTOGENE extended its partnership with Takeda Pharmaceutical Company Limited, a leader in developing therapies for lysosomal storage disorders (LSDs) such as fabry disease. This collaboration ensures continued access to diagnostic testing for patients globally, improving patient access to rapid and accurate diagnostics for LSDs, including fabry disease.
Major companies operating in the fabry disease treatment market are Sanofi S.A., GSK plc, Takeda Pharmaceutical Company Limited, CHIESI Farmaceutici S.p.A., Ultragenyx Pharmaceutical Inc., Amicus Therapeutics Inc., JCR Pharmaceuticals Co. Ltd., Sangamo Therapeutics Inc., Idorsia Pharmaceuticals Ltd., Centogene N.V., Protalix BioTherapeutics, Avrobio Inc., ISU Abxis, Synageva BioPharma Corp., Greenovation Biotech GmbH, CANbridge Pharmaceuticals, Freeline, 4D Molecular Therapeutics, Abeona Therapeutics Inc., GC Biopharma Corporation.
North America was the largest region in the fabry disease treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the fabry disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the fabry disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Fabry disease stems from a deficiency in alpha-galactosidase A, an enzyme crucial for breaking down globotriaosylceramide (Gb3 or GL-3) fat. This results in Gb3 accumulation within cells, prompting symptoms such as pain, kidney issues, heart complications, and strokes. Treatment aims to curtail Gb3 buildup, manage symptoms, slow organ damage, and enhance patients' quality of life.
The fabry disease treatment landscape includes substrate reduction therapy, enzyme replacement therapy, chaperone interventions, among others. Substrate reduction therapy (SRT) targets the reduction of specific substances in the body linked to fabry disease. These therapies can be administered orally or intravenously and cater to varying disease severities such as classic and late-onset fabry disease. Distribution channels encompass hospital, retail, and online pharmacies.
The fabry disease treatment market research report is one of a series of new reports that provides fabry disease treatment market statistics, including the fabry disease treatment industry global market size, regional shares, competitors with fabry disease treatment market share, detailed fabry disease treatment market segments, market trends, and opportunities, and any further data you may need to thrive in the fabry disease treatment industry. These fabry disease treatment market research reports deliver a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The fabry disease treatment market consists of revenues earned by entities by providing services such as diagnosis and screening, enzyme replacement therapy, gene therapy, disease management programs, and patient education and support. The market value includes the value of related goods sold by the service provider or included within the service offering. The Fabry disease treatment market also includes sales of Agalsidase alfa, Agalsidase beta, and Migalastat. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The fabry disease treatment market size has grown strongly in recent years. It will grow from $2.09 billion in 2024 to $2.28 billion in 2025 at a compound annual growth rate (CAGR) of 9.1%. The growth in the historic period can be attributed to increasing awareness and diagnosis rates, increasing adoption of enzyme replacement therapies, increasing use of chaperone therapies, increasing healthcare infrastructure, rise in investment in rare disease research, increasing population, and increasing regulatory incentives for orphan drugs.
The fabry disease treatment market size is expected to see strong growth in the next few years. It will grow to $3.2 billion in 2029 at a compound annual growth rate (CAGR) of 8.8%. The growth in the forecast period can be attributed to increasing prevalence of the disease, rising awareness and diagnosis rates, expanding healthcare infrastructure, growing investment in rare disease research, growing patient awareness, and expanding reimbursement policies. Major trends in the forecast period include advancements in gene therapy, advancements in enzyme replacement therapies, advancements in diagnostic techniques, advancements in novel therapies, and integration of digital health technologies for patient management.
The growth of the Fabry disease treatment market is expected to be driven by the increasing prevalence of renal diseases. Renal diseases, or kidney diseases, encompass conditions that affect kidney function and are on the rise due to genetic factors, lifestyle choices, environmental influences, and underlying health issues. Fabry disease, specifically caused by the buildup of globotriaosylceramide (Gb3) in kidney cells, results in kidney damage and dysfunction. This emphasizes the urgent need for early detection and intervention to prevent kidney-related complications. For instance, recent data from the Australian Bureau of Statistics revealed a 0.2% increase in kidney disease cases over the past decade, with 246,200 people (1% of the population) affected in 2022, highlighting the escalating prevalence of renal diseases and their impact on the fabry disease treatment market.
Key players in the Fabry disease treatment market are actively developing plant cell-based therapies to enhance treatment efficacy, reduce costs, and improve patient outcomes through innovative and sustainable methods. Plant cell-based treatments for fabry disease involve using plant cells to produce deficient enzymes found in individuals with fabry disease. These plant-derived enzymes serve as a therapeutic option to replace the missing or defective enzymes in the body, aiming to alleviate symptoms and enhance patients' overall health. For example, ELFABRIO, an enzyme replacement therapy developed by Chiesi Global Rare Diseases and Protalix BioTherapeutics Inc., was approved in May 2023. ELFABRIO utilizes recombinant human α-Galactosidase-A enzyme produced in plant-cell culture and modified with PEGylation to extend its half-life significantly. This innovative treatment approach reduces treatment frequency, increases convenience, and improves symptom management, ultimately enhancing the quality of life for fabry disease patients.
In March 2024, CENTOGENE extended its partnership with Takeda Pharmaceutical Company Limited, a leader in developing therapies for lysosomal storage disorders (LSDs) such as fabry disease. This collaboration ensures continued access to diagnostic testing for patients globally, improving patient access to rapid and accurate diagnostics for LSDs, including fabry disease.
Major companies operating in the fabry disease treatment market are Sanofi S.A., GSK plc, Takeda Pharmaceutical Company Limited, CHIESI Farmaceutici S.p.A., Ultragenyx Pharmaceutical Inc., Amicus Therapeutics Inc., JCR Pharmaceuticals Co. Ltd., Sangamo Therapeutics Inc., Idorsia Pharmaceuticals Ltd., Centogene N.V., Protalix BioTherapeutics, Avrobio Inc., ISU Abxis, Synageva BioPharma Corp., Greenovation Biotech GmbH, CANbridge Pharmaceuticals, Freeline, 4D Molecular Therapeutics, Abeona Therapeutics Inc., GC Biopharma Corporation.
North America was the largest region in the fabry disease treatment market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the fabry disease treatment market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the fabry disease treatment market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
Fabry disease stems from a deficiency in alpha-galactosidase A, an enzyme crucial for breaking down globotriaosylceramide (Gb3 or GL-3) fat. This results in Gb3 accumulation within cells, prompting symptoms such as pain, kidney issues, heart complications, and strokes. Treatment aims to curtail Gb3 buildup, manage symptoms, slow organ damage, and enhance patients' quality of life.
The fabry disease treatment landscape includes substrate reduction therapy, enzyme replacement therapy, chaperone interventions, among others. Substrate reduction therapy (SRT) targets the reduction of specific substances in the body linked to fabry disease. These therapies can be administered orally or intravenously and cater to varying disease severities such as classic and late-onset fabry disease. Distribution channels encompass hospital, retail, and online pharmacies.
The fabry disease treatment market research report is one of a series of new reports that provides fabry disease treatment market statistics, including the fabry disease treatment industry global market size, regional shares, competitors with fabry disease treatment market share, detailed fabry disease treatment market segments, market trends, and opportunities, and any further data you may need to thrive in the fabry disease treatment industry. These fabry disease treatment market research reports deliver a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The fabry disease treatment market consists of revenues earned by entities by providing services such as diagnosis and screening, enzyme replacement therapy, gene therapy, disease management programs, and patient education and support. The market value includes the value of related goods sold by the service provider or included within the service offering. The Fabry disease treatment market also includes sales of Agalsidase alfa, Agalsidase beta, and Migalastat. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Table of Contents
1. Executive Summary2. Fabry Disease Treatment Market Characteristics3. Fabry Disease Treatment Market Trends and Strategies4. Fabry Disease Treatment Market - Macro Economic Scenario Including the Impact of Interest Rates, Inflation, Geopolitics, and the Recovery from COVID-19 on the Market32. Global Fabry Disease Treatment Market Competitive Benchmarking and Dashboard33. Key Mergers and Acquisitions in the Fabry Disease Treatment Market34. Recent Developments in the Fabry Disease Treatment Market
5. Global Fabry Disease Treatment Growth Analysis and Strategic Analysis Framework
6. Fabry Disease Treatment Market Segmentation
7. Fabry Disease Treatment Market Regional and Country Analysis
8. Asia-Pacific Fabry Disease Treatment Market
9. China Fabry Disease Treatment Market
10. India Fabry Disease Treatment Market
11. Japan Fabry Disease Treatment Market
12. Australia Fabry Disease Treatment Market
13. Indonesia Fabry Disease Treatment Market
14. South Korea Fabry Disease Treatment Market
15. Western Europe Fabry Disease Treatment Market
16. UK Fabry Disease Treatment Market
17. Germany Fabry Disease Treatment Market
18. France Fabry Disease Treatment Market
19. Italy Fabry Disease Treatment Market
20. Spain Fabry Disease Treatment Market
21. Eastern Europe Fabry Disease Treatment Market
22. Russia Fabry Disease Treatment Market
23. North America Fabry Disease Treatment Market
24. USA Fabry Disease Treatment Market
25. Canada Fabry Disease Treatment Market
26. South America Fabry Disease Treatment Market
27. Brazil Fabry Disease Treatment Market
28. Middle East Fabry Disease Treatment Market
29. Africa Fabry Disease Treatment Market
30. Fabry Disease Treatment Market Competitive Landscape and Company Profiles
31. Fabry Disease Treatment Market Other Major and Innovative Companies
35. Fabry Disease Treatment Market High Potential Countries, Segments and Strategies
36. Appendix
Executive Summary
Fabry Disease Treatment Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on fabry disease treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for fabry disease treatment ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The fabry disease treatment market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include: the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Treatment: Substrate Reduction Therapy; Enzyme Replacement Therapy; Chaperone Treatment; Other Treatments2) By Route of Administration: Oral Route; Intravenous Route
3) By Disease Severity: Classic Fabry Disease; Late-Onset Fabry Disease
4) By Distribution Channel: Hospital Pharmacies; Retail Pharmacies; Online Pharmacies
Subsegments:
1) By Substrate Reduction Therapy: Migalastat Hydrochloride (Galafold); Other Substrate Reduction Therapies2) By Enzyme Replacement Therapy: Agalsidase Beta (Fabrazyme); Agalsidase Alfa (Replagal); Other Enzyme Replacement Therapies
3) By Chaperone Treatment: Migalastat Hydrochloride (Galafold)
4) By Other Treatments: Gene Therapy; Symptomatic Treatment
Key Companies Mentioned: Sanofi S.a.; GSK plc; Takeda Pharmaceutical Company Limited; CHIESI Farmaceutici S.p.a.; Ultragenyx Pharmaceutical Inc.
Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
The major companies featured in this Fabry Disease Treatment market report include:- Sanofi S.A.
- GSK plc
- Takeda Pharmaceutical Company Limited
- CHIESI Farmaceutici S.p.A.
- Ultragenyx Pharmaceutical Inc.
- Amicus Therapeutics Inc.
- JCR Pharmaceuticals Co. Ltd.
- Sangamo Therapeutics Inc.
- Idorsia Pharmaceuticals Ltd.
- Centogene N.V.
- Protalix BioTherapeutics
- Avrobio Inc.
- ISU Abxis
- Synageva BioPharma Corp.
- Greenovation Biotech GmbH
- CANbridge Pharmaceuticals
- Freeline
- 4D Molecular Therapeutics
- Abeona Therapeutics Inc.
- GC Biopharma Corporation
Table Information
Report Attribute | Details |
---|---|
No. of Pages | 200 |
Published | April 2025 |
Forecast Period | 2025 - 2029 |
Estimated Market Value ( USD | $ 2.28 Billion |
Forecasted Market Value ( USD | $ 3.2 Billion |
Compound Annual Growth Rate | 8.8% |
Regions Covered | Global |
No. of Companies Mentioned | 21 |