The Fabry disease treatment market was valued at USD 2.75 Billion in 2024 driven by innovations in treatment options across the 8 major markets. It is expected to grow at a CAGR of 9.90% during the forecast period of 2025-2034 and attain a market value of USD 7.07 Billion by 2034.
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The market is expected to grow due to increasing gene therapy innovations, offering more effective and personalised treatment options for patients with rare genetic disorders.
With advancements in enzyme replacement therapies and gene-based treatments, the market is poised for expansion, addressing unmet needs and improving the quality of life for Fabry disease patients.
The increasing adoption of orphan drug designations and regulatory approvals for novel therapies will drive market growth, attracting more investment and fostering further advancements in treatment options.
Fabry disease is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. This enzyme is crucial for breaking down lipids in the body. Without it, harmful levels of lipids accumulate in cells, leading to severe health problems such as kidney failure, heart issues, strokes, and skin conditions, affecting various organs over time.
Advent of Advanced Treatments and Genetic Therapy Innovation to Accelerate Market Expansion
The market is witnessing growth driven by advancements in gene therapy and the rising need for effective treatments for rare diseases. For instance, in December 2024, Exegenesis Bio received an orphan drug designation from the FDA for EXG110, a novel gene therapy to treat Fabry disease. This therapy works by delivering a genetic payload directly to liver and heart cells, offering a potentially more effective, safer, and easier one-time treatment. Exegenesis Bio's progress in China, along with plans for a US-based clinical trial, is expected to enhance the market's development, offering new hope to patients. The approval of EXG110 could boost market value, particularly in regions where alternative therapies are limited, driving growth in the forecast period.
Rising Focus on Rare Disease Treatments and FDA Approvals to Meet the Growing Fabry Disease Treatment Market Demand
The increasing focus on rare disease therapies and the successful development of innovative gene therapies propel the growth of the market. For instance, in September 2024, uniQure N.V. received an FDA orphan drug designation for its investigational gene therapy, AMT-191, targeting Fabry disease. AMT-191 is currently undergoing Phase I/IIa trials in the US, with the first patient having been dosed in August 2024. This development is poised to have a significant impact on the market by introducing a potential breakthrough treatment for Fabry disease. With a promising clinical trial underway, AMT-191 could open doors to new treatment avenues, potentially expanding the market and enhancing therapeutic options in the forecast period.
The market is witnessing several trends and developments to improve the current scenario. Some of the notable trends are as follows:
The market report offers a detailed analysis of the market based on the following segments:
Market Breakup by Treatment
Market Breakup by Route of Administration
Market Breakup by Distribution Channel
Market Breakup by Region
Enzyme Replacement Therapy to Lead the Market Share by Treatment
Enzyme Replacement Therapy (ERT) is expected to hold the largest share in the Fabry disease treatment market due to its established efficacy in managing the disease. ERT provides long-term symptom relief by replacing the deficient enzyme, alpha-galactosidase A. Its widespread adoption, along with proven clinical success, continues to drive significant market demand compared to other treatment options.
Regionally, the market report offers insights into the United States, United Kingdom, Germany, France, Italy, Spain, Japan and India. Among these, the United States holds the largest market share due to its well-established healthcare infrastructure, high treatment adoption rates, and significant investment in rare disease therapies. Additionally, the presence of leading pharmaceutical companies and robust regulatory support drive the demand for advanced treatments in the country.
The key features of the market report comprise patent analysis, clinical trials analysis, grants analysis, funding and investment analysis, and strategic initiatives by the leading players. The major companies in the market are as follows:
Headquartered in Paris, France, Sanofi SA was established in 2004. The company is a global leader in healthcare, with a strong presence in the Fabry disease treatment market. Sanofi’s portfolio includes enzyme replacement therapies such as Fabrazyme, which is approved for the treatment of Fabry disease, addressing unmet medical needs with innovative therapies for rare genetic conditions. In February 2023, Sanofi Speciality Care reaffirmed its commitment to improving the lives of patients with rare diseases in India. Over the past 40 years, the company has launched therapies for conditions like Gaucher, Fabry, Pompe, Mucopolysaccharidosis I, and Acid Sphingomyelinase deficiency (ASMD). Sanofi is also set to introduce two new therapies, Nexviazyme and Xenpozyme, for Pompe and Niemann-Pick disease, following orphan drug status and approval in several countries. These efforts highlight Sanofi's leadership in the rare disease treatment space.
M6P Therapeutics Inc., founded in 2020, is a biotechnology company based in the United States. The company is focused on developing gene therapies for rare genetic diseases, including Fabry disease. M6P's flagship product aims to correct the underlying genetic defect in Fabry patients, offering a potential one-time treatment that could revolutionise current approaches to disease management.
Takeda Pharmaceutical Company Limited, founded in 1781 and headquartered in Osaka, Japan, is a global biopharmaceutical leader. In the Fabry disease treatment space, Takeda offers Replagal, an enzyme replacement therapy designed to replace the deficient alpha-galactosidase A enzyme in patients with Fabry disease. The company is dedicated to addressing rare and complex diseases with advanced, patient-centric therapies. In March 2024, Takeda extended its collaboration with Centogene to enhance diagnostic services for lysosomal storage disorders, including Fabry disease. The partnership, initially launched to improve access to fast and reliable diagnostics for rare diseases like Gaucher and Hunter syndrome, will continue to support patients in receiving accurate diagnoses. This extended collaboration strengthens Takeda’s commitment to improving patient outcomes and advancing the treatment landscape for lysosomal storage disorders, driving innovation in the rare disease space.
Founded in 1926, Yuhan Corporation is a South Korean pharmaceutical company headquartered in Seoul. The company has been expanding its portfolio to include treatments for rare diseases like Fabry disease. Yuhan collaborates with global partners to develop innovative therapies, including enzyme replacement treatments aimed at improving outcomes for patients suffering from Fabry disease and other lysosomal storage disorders.
*Please note that this is only a partial list; the complete list of key players is available in the full report. Additionally, the list of key players can be customized to better suit your needs.*
Other key players in the market include Freeline Therapeutics Holdings PLC, Chiesi Farmaceutici S.p.A., Protalix BioTherapeutics Inc., JCR Pharmaceuticals Co., Ltd., Amicus Therapeutics, Inc., and ISU ABXIS.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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| REPORT FEATURES | DETAILS |
| Base Year | 2024 |
| Historical Period | 2018-2024 |
| Forecast Period | 2025-2034 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Treatment |
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| Breakup by Route of Administration |
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| Breakup by Distribution Channel |
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| Breakup by Region |
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| Market Dynamics |
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| Supplier Landscape |
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| Companies Covered |
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United States (Head Office)
30 North Gould Street, Sheridan, WY 82801
+1-415-325-5166
Australia
63 Fiona Drive, Tamworth, NSW
+61-448-061-727
India
C130 Sector 2 Noida, Uttar Pradesh 201301
+91-723-689-1189
Philippines
40th Floor, PBCom Tower, 6795 Ayala Avenue Cor V.A Rufino St. Makati City, 1226.
+63-287-899-028, +63-967-048-3306
United Kingdom
6 Gardner Place, Becketts Close, Feltham TW14 0BX, Greater London
+44-753-713-2163
Vietnam
193/26/4 St.no.6, Ward Binh Hung Hoa, Binh Tan District, Ho Chi Minh City
+84-865-399-124
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