Fabry Disease Treatment Market - By Treatment, By Route of Administration, By End Use - Global Forecast 2025 – 2034

Fabry Disease Treatment Market Size

The global Fabry disease treatment market size was valued at USD 2.3 billion in 2024. The market is expected to grow from USD 2.5 billion in 2025 to USD 5.1 billion in 2032, at a CAGR of 8.2% during the forecast period. The rising demand for efficient, precise, highly sensitive and specific Fabry disease treatments is expected to spur business growth.
 

The rising cases of Fabry and associated complications with the disease are expected to increase the demand for advanced Fabry disease treatments across the globe. For instance, as per estimates from the Fabry Disease News reports in May 2024, there is evidence of a later appearance of Fabry disease which appears to be relatively common, occurring in approximately 1 in 1000 to 3000 males, and 1 in 6000 to 40,000 females.
 

However, the classic type of Fabry Disease is rare and is estimated to affect approximately 1 in 22,000 to 40,000 males. Thus, the increasing prevalence of Fabry disease coupled with growing investment by major players in the market for the development of more effective and superior treatment, results in the launch of novel therapies and expanded availability to meet the increased demand for the precise treatment of this rare genetic illness internationally propel market growth during the analysis period.
 

The market is further strengthened by the increasing awareness and early diagnosis of disease. In addition, the global aging population is more susceptible to genetic diseases, including Fabry. As life expectancy increases, the number of people that might develop Fabry disease also increases, which can increase the demand for treatments thereby contributing to market growth. Moreover, numerous governments and non-profit organizations comprising Rare Diseases International (RDI) are operating to create awareness regarding the management of such rare genetic disorders among the population. These significant efforts will positively impact on the demand for related treatment, thereby boosting the business revenue.
 

Fabry disease is an uncommon hereditary disorder that has a notable impact on several organs of the body such as the kidneys, heart, and skin. Episodes of pain especially at the lower limbs and feet (known as acroparesthesias), small, dark red lesions on the skin termed “angiokeratomas” and decrease in sweating (hypohidrosis) cloudiness or streaks in the front part of the eye (corneal opacity or corneal verticillate” are very common. It is caused by the accumulation of a particular fat called globotriaosylceramide (GL-3) within the tissues of the body. The most specific and effective treatment available is enzyme replacement therapy and chaperone therapy.
 

Fabry Disease Treatment Market Trends

The increasing government initiatives and funding for the development of innovative therapies contribute to the growth of the market.
 

• Government financing and favourable policies have led to increased funding and R&D on rare diseases such as Fabry’s disease. For instance, in March 2023, the Canadian Minister of Health announced new steps to implement a National Strategy for Drugs for Rare Diseases with funding of up to USD 1.5 billion over three years.
   
• Thus, increasing research and development activities with significant improvements in treatment modalities, including the development of advanced therapies such as venglustat (Ibiglustat), gene therapy (ST-920) will drive the growth of the market during the analysis period.
   
• Furthermore, the key players operating in the market are continuously involved in research and development activities and investing heavily for the development of the development of novel treatment options, thereby contributing the market growth.
   
• For example, Protalix BioTherapeutics announces USD 43.7 million in financing to further advance its programs in Fabry disease. Therefore, the significant investment from some of the leading investors in Israel and the U.S. emphasizes Protalix's allegiance to bringing important treatment to the Fabry patient.
   
• In addition, regulatory bodies like the U.S. FDA and the European Medicines Agency (EMA) as well as Japan’s PDMA provided orphan drug status to several candidate Fabry disease drugs. For instance, Exegenesis Bio’s novel gene therapy candidate for Fabry disease, EXG110, received orphan drug designation from the U.S. FDA in December 2024.
   
• Therefore, the demand for treatment rises with the increasing recognition of Fabry disease by healthcare practitioners as well as patients due to the less restrictive advanced treatment options available. 
   

Fabry Disease Treatment Market Analysis

Based on treatment, the global Fabry disease treatment market is divided into enzyme replacement therapy (ERT), chaperone treatment, and other treatment types. The market was valued at USD 2.2 billion in 2023. The enzyme replacement therapy (ERT) segment dominated the market and was valued at USD 1.7 billion in 2024.
 

• The growth of enzyme replacement therapy (ERT) segment is high owing to rising prevalence of disease that directly boosts the demand for ERT, as it is the standard treatment for Fabry disease to replace the deficient enzyme alpha-galactosidase A.
   
• Additionally, the advancement in diagnostic techniques such as next-generation sequencing and enzyme assays have led to earlier and more accurate diagnosis of Fabry disease, contributing to the growth of the ERT market. As an example, Centogene has extended a collaboration deal with Takeda in which the two companies will continue to provide diagnostic services to patients with lysosomal storage disorders which may help people with Fabry disease to get a correct diagnosis.
   
• Moreover, the wide availability of these drugs in pharmacies further propels the overall market growth. In addition, regulatory approvals for novel ERT formulations and delivery methods, such as Pegunigalsidase alfa (PRX-102), have expanded treatment options. These advancements are often coupled with improved efficacy and reduced infusion-related reactions, making ERT more acceptable to patients. have fueled growth in the drugs segment.
   
• Furthermore, the introduction of plant-based recombinant ERT and personalized treatment options improve the patient outcome, thereby companies are investing in research to minimize adverse effects related to infusion and optimize the dosage regimen.
   

Based on the route of administration, the global Fabry disease treatment market is categorized into intravenous and oral. The intravenous segment accounted for the highest market share of 68.3% in 2024 and is anticipated to witness growth at a CAGR of 8.1% during the analysis period.
 

• An increase in the diagnosed Fabry disease population due to better awareness and genetic screening programs is boosting the demand for intravenous therapies.
   
• Moreover, clinical trials have consistently shown the superiority of intravenous ERT in stabilizing Fabry disease symptoms over long durations. For instance, in 2021, a multicenter trial reported that Fabrazyme-treated patients had a 55% reduction in cardiac events and a 40% improvement in kidney function over five years, that boosts market growth.
   
• Moreover, the availability of advanced healthcare infrastructure in developed markets, such as North America and Europe, supports the growth of IV administration. These regions have a higher concentration of Fabry disease patients receiving ERT, thereby fostering the overall market progression.
   
• Furthermore, the advancement infusion technique with the introduction of home-infusion services improve patient outcome and convenience. In addition, the growing patient compliance with twice a week administration of ERT via IV route in homecare settings reduces hospital visits, raise the adoption of drug through IV route, contributes to the market growth.  
   

Based on the end use, the global Fabry disease treatment market is categorized into hospitals, homecare settings, and other end users. The hospitals segment dominated the market in 2024 and is anticipated to reach USD 2.9 billion by 2034 with a significant CAGR of 8% during the analysis period.
 

• Hospitals are increasingly equipped with advanced diagnostic tools such as genetic testing and enzyme activity assays, which are essential for the accurate diagnosis of Fabry disease. The early and precise diagnosis of disease increases the demand for effective treatment options thereby drives the growth of the market.
   
• Additionally, hospitals are the primary providers of enzyme replacement therapy (ERT), the cornerstone of Fabry disease treatment. The complexity of ERT administration and the need for monitoring drive patients to hospital settings.
   
• Moreover, Fabry disease impacts multiple organ systems, including the kidneys, heart, and nervous system, necessitating multidisciplinary care. Hospitals are uniquely positioned to provide comprehensive care involving nephrologists, cardiologists, neurologists, and genetic counselors, driving market growth.
   
• Hospitals are playing a growing role in clinical trials through collaborations with research organizations, which supports the development and introduction of novel therapies for patients. Additionally, the establishment of specialized treatment centers, along with the increasing number of hospitals providing dedicated care for Fabry disease, is contributing significantly to market growth.
   

In 2024, the U.S. held a significant position in the North America Fabry disease treatment market and was valued at USD 945.8 million from USD 878.7 million in 2023.
 

• This market growth is owing to the favorable government initiatives, increasing prevalence of Fabry disease, the presence of key industry players, an upsurge in the number of treatment procedures in U.S.
   
• For example, as per the report published by National Fabry Disease Foundation, about 50,000 people in the U.S. live with Fabry disease including classic and late-onset forms of the disease.
   
• Moreover, the enzyme replacement therapy (ERT) and chaperone therapy has become a significant option for the management of complications associated with this target ailment, thereby stimulating the treatment demand positively in the country.
   
• Furthermore, the presence of prominent industry players such as Takeda, Sanofi, and Amicus Therapeutics among others in the region are engaged in new therapies development and commercialization that facilitates market expansion in the U.S.
   
• The government and private organization raises awareness program for Fabry disease, this leads in increasing number of people diagnosed with disease raises the demand for effective treatment, thereby contributing to the market growth.
   
• For instance, in May 2024, the American Kidney Fund (AKF) and Sanofi announced an awareness campaign to increase visibility of Fabry disease. The campaign will include encouraging patients with chronic kidney disease (CKD) with an unknown underlying cause to get tested for Fabry disease.
   

Germany shows strong growth potential in the Europe Fabry disease treatment market.
 

• Germany has one of the most developed healthcare systems in Europe, with high levels of accessibility and advanced facilities. This supports early diagnosis and effective management of rare disease treatment like Fabry disease, contributing to the overall market growth.
   
• Moreover, Germany is home to research hubs like the German Center for Rare Diseases (DZNE), which collaborates on Fabry disease studies. Clinical trials conducted in Germany for novel therapies, including gene therapies, have attracted significant investment.
   
• Additionally, Germany has been a leader in adopting novel therapies, including enzyme replacement therapy (ERT) and chaperone therapies, which are the primary treatments for Fabry disease. The increasing availability of advanced drugs, including gene therapies under development, fuels market growth.
   
• For example, in August 2021, Amicus Therapeutics announced that the European Commission has approved Galafold (migalastat) for use in adolescents aged 12 to 16 years weighing ≥ 45 kg with a confirmed diagnosis of Fabry disease. This helped the company expand its sales in Europe.
   

China Fabry disease treatment market is expected to grow significantly in Asia Pacific market during the analysis period.
 

• China's healthcare infrastructure is rapidly improving, particularly in urban areas, increasing access to advanced treatments for rare diseases like Fabry disease.
   
• Moreover, China has witnessed approvals of enzyme replacement therapies (ERT) and oral chaperone therapies for Fabry disease, providing patients with more treatment options.
   
• The Chinese government has been actively involved in supporting the treatment of rare diseases, including Fabry disease. Initiatives such as the inclusion of certain rare disease treatments in national health insurance schemes have made therapies more accessible and affordable, thereby stimulating market growth.
   
• In addition, the local players in China are continuously involved in research and development activities for the manufacturing of novel drug molecules. For example, in August 2023, AceLink Therapeutics opened its first clinical site in China for a Phase 2 clinical trial testing AL01211 as a treatment for Fabry disease. The trial is actively screening and enrolling patients across six sites in China, including Shanghai’s Ruijin hospital.
   

Latin America: Brazil's Fabry disease treatment market is projected to witness growth in coming years.
 

• The Brazilian government has implemented policies to ensure treatment access for rare diseases. Under the National Policy for Comprehensive Care for People with Rare Diseases (2014), Fabry disease is recognized as requiring specialized care.
   
• Furthermore, pharmaceutical companies are increasingly focusing on Brazil as an emerging market for rare disease treatments. Leading drugs for Fabry disease, such as Fabrazyme (agalsidase beta) and Replagal (agalsidase alfa), are marketed in the country.
•  
• Additionally, organizations such as the Brazilian Fabry Disease Association (ABRAF) play a vital role in raising awareness, advocating for better patient care, and ensuring treatment availability.
   

Saudi Arabia is anticipated to grow in the Middle East and Africa Fabry disease treatment market.
 

• The Saudi government has invested heavily in healthcare through its Vision 2030 initiative, which aims to improve the healthcare system. In addition, Saudi Arabia's growing focus on genetic screening programs helps identify Fabry disease early, allowing for timely treatment interventions thereby boosting the market for Fabry disease treatment.
   
• Moreover, Saudi Arabia’s government provides significant subsidies for treating rare genetic diseases, including Fabry disease, under its universal healthcare system. High-cost treatments are often covered under government programs, reducing financial barriers for patients.
   

Fabry Disease Treatment Market Share

The top 5 players such as Amicus Therapeutics, Novartis, Pfizer, Sanofi, and Takeda Pharmaceuticals accounts for approximately 60% of the market share in 2024. These players focus on various strategies such as acquisition, business expansion, research and development activities and novel product launches to consolidate their market presence.
 

For instance, in February 2022, Takeda Pharmaceutical Company and Sumitomo Dainippon Pharma, announced that Takeda succeed the manufacturing and marketing approval and the marketing rights of REPLAGAL 3.5 mg for Fabry disease, an α-galactosidase enzyme intravenous (IV) infusion from Sumitomo Dainippon Pharma. Established pharmaceutical giants offer a well-known Fabry disease treatment under various brand names.
 

Key players are engaging in strategic initiatives such as collaboration, product launch, investment, and partnerships to strengthen their product portfolio. The companies are actively adopting a multi-pronged approaches to address the rising demand for Fabry disease treatment. Furthermore, the market is highly consolidated with few players operating in the market. Hence, leading players are constantly adopting key strategies for the growth of the market.
 

Fabry Disease Treatment Market Companies

Prominent players operating in the Fabry disease treatment industry are as mentioned below:

• Amicus Therapeutics
• Avrobio
• Freeline Therapeutics
• Idorsia Pharmaceuticals
• ISU Abxis
• JCR Pharmaceuticals
• Novartis
• Pfizer
• Protalix BioTherapeutics
• Sanofi SA
• Takeda Pharmaceuticals
• Viatris
   
• Many leading players such as Sanofi focuses on product approval, mergers and acquisitions to gain a competitive edge in the market. For instance, in May 2021, Sanofi has been granted Orphan Drug Designation for venglustat in Europe and the U.S. for treatment of patients with Fabry disease, ADPKD, Gaucher disease and GM2 gangliosidosis. Companies are getting orphan drug designation to introduce more products for Fabry disease in the market.
   

Fabry Disease Treatment Industry News

• In November 2023, uniQure announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for AMT-191, the Company’s gene therapy candidate for Fabry disease. AMT-191 comprises an AAV5 vector that delivers a -galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein. This helped the company expand its product range.
   
• In May 2023, Chiesi Global Rare Diseases, announced that the U.S. Food and Drug Administration (FDA) has approved PRX-102 (pegunigalsidase alfa-iwxj) in the U.S. for the treatment of adult patients with Fabry disease. This product approval helped the company expand its product range in U.S. market.
   
• In May 2023, Sangamo Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. This approval aimed to broaden the treatment availability, complementing existing therapies for Fabry disease.
   

The Fabry disease treatment market research report includes in-depth coverage of the industry with estimates and forecast in terms of revenue in USD Million from 2021 – 2034 for the following segments:

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Market, By Treatment

• Enzyme replacement therapy (ERT)
• Chaperone treatment
• Other treatment types

Market, By Route of Administration

• Intravenous
• Oral

Market, By End Use

• Hospitals
• Homecare settings
• Other end users

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Spain
  • Italy
  • Netherlands
• Asia Pacific
  • China
  • India
  • Japan
  • Australia
  • South Korea 
• Latin America
  • Brazil
  • Mexico
  • Argentina 
• Middle East and Africa
  • South Africa
  • Saudi Arabia
  • UAE