Fabry Disease Market

Market Overview:

The 7 major fabry disease markets reached a value of USD 1,637.7 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 3,898.0 Million by 2035, exhibiting a growth rate (CAGR) of 8.20% during 2025-2035.

The fabry disease market has been comprehensively analyzed in IMARC's new report titled "Fabry Disease Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Fabry disease is an X-linked, rare lysosomal storage disease due to mutations in the GLA gene that lead to a deficiency of the alpha-galactosidase A enzyme. This enzyme deficiency causes globotriaosylceramide (Gb3) to accumulate in lysosomes and result in progressive damage to several organ systems. The illness comes with a wide range of symptoms such as neuropathic pain, angiokeratomas, gastrointestinal upset, hypohidrosis, opacities of the cornea, and life-threatening complications like renal failure, cardiomyopathy, and stroke. The disease severity is determined by levels of enzyme activity, which has the classic variant diagnosed in childhood and the adult-onset variant diagnosed in adulthood. Fabry disease is diagnosed by enzyme activity assays in men, genetic evaluation for GLA mutations, and biomarker study (lyso-Gb3 levels) for confirmation of the presence of disease in both genders. Diagnosis earlier and correctly helps in the treatment at the proper time and to have better patients' outcomes.

The Fabry disease market is witnessing tremendous growth as a result of increasing awareness, improvements in diagnostic methods, and the growing use of new treatment modalities. Enzyme replacement therapies (ERTs) such as agalsidase alfa and agalsidase beta are still the mainstay of treatment, significantly lowering Gb3 accumulation and halting disease progression. Yet, their lifelong intravenous use and the risk of immune reactions have fueled the need for other therapies. Chaperone therapy, such as migalastat, has become an effective oral form of treatment in patients with amenable mutations with increased patient compliance. Next-generation therapies, namely substrate reduction therapies (SRTs) and gene therapies, are also on the horizon in clinical trials as possible long-term treatments. Gene therapies have been developed to achieve normal enzyme activity via single doses, which might revolutionize treatment of Fabry disease. In spite of these advances, the high cost of treatment, restricted access to therapies in some areas, and delays in diagnosis due to the heterogeneous presentation of the disease are the major challenges. Overcoming these obstacles through increased newborn screening programs, enhanced physician education, and increased patient access to innovative therapies will be critical to continued market growth.

IMARC Group's new report provides an exhaustive analysis of the fabry disease market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the fabry disease market in any manner.

Recent Developments:

• In March 2024, 4D-310 gene therapy improved cardiac function in Fabry disease patients, with benefits sustained for two years. No major adverse events occurred in 33 months of follow-up.
• In January 2024, Sangamo Therapeutics continued its Fabry disease program despite Pfizer ending a hemophilia A collaboration, targeting a Biologics License Application (BLA) submission in late 2025.

Key Highlights:

• Fabry disease affects 1 in 40,000 to 1 in 117,000 live births globally, with potentially higher prevalence in certain regions like India.
• X-linked disorder; sons of carrier mothers have a 50% chance of inheritance, while daughters have a 50% chance of being carriers.
• Classic Fabry symptoms appear in childhood, including burning pain and skin lesions, while late-onset cases emerge in the 30s or 40s.
• Males live about 58 years (vs. 75 general population), females about 75 years (vs. 80 general population), with organ complications affecting survival.
• Lipid accumulation causes kidney failure, heart disease, and strokes, increasing risks of cardiovascular and renal complications over time.

Drugs:

Fabrazyme (agalsidase beta) is recombinant human α-galactosidase enzyme replacement therapy for the treatment of Fabry disease. It breaks down accumulated glycosphingolipids within lysosomes and decreases globotriaosylceramide (GL-3) levels in principal organs. Given intravenously every other week at 1 mg/kg, Fabrazyme manages and retards the progression of the disease.

Elfabrio (pegunigalsidase alfa) is an adult treatment for Fabry disease using enzyme replacement therapy. Given every other week by intravenous infusion, it provides deficient α-galactosidase-A, lowering toxic globotriaosylceramide (Gb3) levels. Protalix BioTherapeutics and Chiesi Global Rare Diseases developed Elfabrio, which gained approval from the FDA in May 2023.

Galafold (migalastat) is the first oral precision therapy that received approval for treating adults with Fabry disease having amenable GLA mutations. It stabilizes the body's own abnormal enzyme to minimize the accumulation of GL-3. Side effects experienced by many people include headache, nasopharyngitis, urinary tract infection, nausea, and fever.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the fabry disease market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the fabry disease market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current fabry disease marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the fabry disease market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the fabry disease market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the fabry disease market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035​) of fabry disease across the seven major markets?
• What is the number of prevalent cases (​2019-2035) of fabry disease by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of fabry disease by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035​) with fabry disease across the seven major markets?
• What is the size of the fabry disease patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of fabry disease?
• What will be the growth rate of patients across the seven major markets?

Fabry Disease: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for fabry disease drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the fabry disease market?
• What are the key regulatory events related to the fabry disease market?
• What is the structure of clinical trial landscape by status related to the fabry disease market?
• What is the structure of clinical trial landscape by phase related to the fabry disease market?
• What is the structure of clinical trial landscape by route of administration related to the fabry disease market?