PUBLISHER: The Business Research Company | PRODUCT CODE: 1760532
PUBLISHER: The Business Research Company | PRODUCT CODE: 1760532
Fabry disease is an uncommon genetic condition resulting from a shortage of the enzyme alpha-galactosidase A, which causes fat to accumulate in the cells and organs of the body. Treatment aims to alleviate symptoms, prevent further organ damage, and enhance the quality of life for those affected.
Fabry disease has several forms, including types 1, 2, and others. Type 1 Fabry disease, a rare genetic disorder that typically starts in childhood, is caused by a near-total absence of the alpha-galactosidase A enzyme, leading to the accumulation of fat in cells and organs. Treatment options include enzyme replacement therapy, oral medications, adjunct therapies, and additional methods. Diagnosis is achieved through various techniques, including blood tests, genetic testing, parenteral tests, and other diagnostic approaches. Distribution channels for treatment include hospital pharmacies, online pharmacies, and retail pharmacies, serving different end users such as hospitals, homecare providers, specialty clinics, and other healthcare facilities.
The fabry disease market research report is one of a series of new reports from The Business Research Company that provides fabry disease market statistics, including the fabry disease industry global market size, regional shares, competitors with the fabry disease market share, detailed fabry disease market segments, market trends, and opportunities, and any further data you may need to thrive in the fabry disease industry. This fabry disease market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.
The fabry disease market size has grown strongly in recent years. It will grow from $2.10 billion in 2024 to $2.28 billion in 2025 at a compound annual growth rate (CAGR) of 8.2%. The growth observed in the historical period can be credited to factors such as heightened awareness and diagnosis of rare diseases, the development of specialized healthcare infrastructure, government incentives and orphan drug designations, increased funding for rare disease research, and the initial introduction of therapies.
The fabry disease market size is expected to see strong growth in the next few years. It will grow to $3.10 billion in 2029 at a compound annual growth rate (CAGR) of 8.0%. The growth in the forecast period is expected to be driven by the expansion of newborn screening programs, regulatory support for orphan and rare disease treatments, broader healthcare access, the rise of telemedicine and remote care, and a growing demand for personalized medicine. Key trends during this period include the adoption of enzyme replacement therapy, advancements in genetic testing, the integration of AI and digital tools for early detection, AI-powered diagnostic algorithms, and progress in gene therapy.
The increasing emphasis on personalized medicine is anticipated to drive the growth of the Fabry disease market in the future. Personalized medicine tailors treatments based on an individual's genetic makeup, environment, and lifestyle, which improves treatment outcomes. This approach enhances treatment effectiveness and reduces side effects by customizing therapies to meet specific patient needs. Personalized medicine enables the development of treatment plans that consider a patient's unique genetic mutation and disease severity, resulting in better outcomes and fewer adverse effects. For example, in February 2024, the Personalized Medicine Coalition, a U.S.-based non-profit, noted that the FDA approved 16 new personalized treatments for rare disease patients in 2023, a significant rise from six approvals in 2022. As a result, the growing emphasis on personalized medicine is a key factor driving the expansion of the Fabry disease market.
Major companies in the Fabry disease market are focusing on innovative therapies, such as PEGylated enzyme replacement therapy (ERT), to improve treatment outcomes, enhance patient adherence, and reduce immune system reactions linked to traditional ERT. PEGylated ERT is a modified version of standard ERT, where polyethylene glycol (PEG) is added to the enzyme to improve stability, extend its duration in the body, and minimize immune responses. For example, in May 2023, Chiesi Global Rare Diseases, part of the Chiesi Group, announced that the U.S. Food and Drug Administration (FDA) approved PRX-102 (pegunigalsidase alfa) for the treatment of adult Fabry disease patients. The approval was supported by a comprehensive clinical program involving over 140 patients and long-term follow-up of up to 7.5 years, demonstrating the therapy's effectiveness in reducing kidney Gb3 deposits and maintaining kidney function, with a favorable safety profile.
In June 2022, Kyorin, a Japan-based pharmaceutical company, acquired a license from CellGenTech to develop and commercialize a treatment for Fabry disease. With this acquisition, Kyorin intends to leverage CellGenTech's genetically modified human adipocytes (GMAC) technology to create a new treatment for Fabry disease. CellGenTech Inc. is a biotechnology company from Japan that specializes in ex vivo gene therapy solutions for rare diseases, including Fabry disease.
Major players in the fabry disease market are Sanofi S.A., GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, JCR Pharmaceuticals Co. Ltd., Sumitomo Pharma Co. Ltd, Chiesi Farmaceutici S.p.A., Amicus Therapeutics Inc., Idorsia Pharmaceuticals Ltd, Sangamo Therapeutics Inc., uniQure N.V., Protalix BioTherapeutics Inc., Spur Therapeutics Inc., Exegenesis Bio Inc., ISU ABXIS Co. Ltd., Eleva GmbH, AceLink Therapeutics Inc., 4D Molecular Therapeutics Inc., iBio Inc., Freeline Therapeutics Holdings plc, Greenovation Biotech GmbH.
North America was the largest region in the fabry disease market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in fabry disease report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the fabry disease market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The fabry disease market consists of revenues earned by entities by providing services such as genetic counseling, patient support programs, clinical trials and research services, and disease monitoring and management services. The market value includes the value of related goods sold by the service provider or contained within the service offering. Only goods and services traded between entities or sold to end consumers are included.
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 and 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.
Fabry Disease Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses on fabry disease 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.
Where is the largest and fastest growing market for fabry disease ? 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 market global report from the Business Research Company 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 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.