Fabry Disease Treatment Market Analysis – Growth Drivers and Projections

The global Fabry disease treatment market is valued at USD 2.45 billion in 2025 and is expected to reach USD 4.87 billion by 2034, expanding at a CAGR of 7.93% during the forecast period. Growth is fueled by the rising prevalence of Fabry disease and the increasing demand for advanced and effective treatment options.

Last Updated : 01 Aug 2025  |  Report Code : 4404  |  Category : Healthcare   |  Format : PDF / PPT / Excel

List of Contents

  • Last Updated : 01 Aug 2025
  • Report Code : 4404
  • Category : Healthcare

Fabry Disease Treatment Market Size and Companies

The global fabry disease treatment market size was valued at USD 2.27 billion in 2024 and is anticipated to reach around USD 4.87 billion by 2034, growing at a CAGR of 7.93% from 2025 to 2034. The global Fabry disease treatment market is poised for growth due to the escalating prevalence of this condition.

Fabry Disease Treatment Market Size 2025 to 2034

Fabry Disease Treatment Market Key Takeaways

  • The global fabry disease treatment market was valued at USD 2.27 billion in 2024.
  • It is projected to reach USD 4.87 billion by 2034.
  • The market is expected to grow at a CAGR of 7.93% from 2025 to 2034.
  • The North America fabry disease treatment market size accounted for USD 710 million in 2024 and is expected to attain around USD 1,570 million by 2034, poised to grow at a CAGR of 8.25% between 2025 and 2034.
  • North America dominated the market in 2024.
  • Asia Pacific is expected to host the fastest-growing market over the forecast period.
  • By treatment, the enzyme replacement therapy segment dominated the market in 2024.
  • By treatment, the substrate reduction therapy segment grows at a rapid pace in the market over the forecast period.

What is the Role of AI in the Fabry Disease Treatment Market?

Different artificial intelligence (AI) models can transform Fabry disease diagnosis, help monitor progression in affected organs and potentially contribute to personalized therapy development. In diagnostics, AI algorithms can analyze medical images like CT scans, X-rays, and MRIs, identifying subtle anomalies that may be missed by the human eye. This aids in the early detection of diseases like cancer and allows timely interventions to improve patient outcomes. AI improves diagnostics accuracy by identifying patterns in medical images, predicting disease risk, and interpreting high amounts of patient data, may faster and more precisely than human professionals.

AI algorithms analyze medical data to assist doctors in making improved diagnoses by studying patient histories, lab results, and genetic information. By identifying patterns and correlations within the data, AI improves diagnostic accuracy and allows personalized treatment planning.

U.S. Fabry Disease Treatment Market Size and Growth 2025 to 2034

The U.S. fabry disease treatment market size was estimated at USD 540 million in 2024 and is predicted to be worth around USD 1,200 million by 2034 with a CAGR of 8.31% from 2025 to 2034.

U.S. Fabry Disease Treatment Market Size 2025 to 2034

North America dominated the Fabry disease treatment market in 2023. The Fabry disease treatment market in the region is growing due to the higher adoption of novel therapies, advanced healthcare facilities, and favorable reimbursement policies. Health insurance programs covering expensive medications like Fabrazyme and supportive government healthcare policies are encouraging drug companies to invest more in R&D for rare diseases. Additionally, government initiatives to fund and support rare disease treatments are expected to drive market growth throughout the study period further.

  • In May 2024, the American Kidney Fund (AKF) started a complete education and awareness effort through an initiative to make Fabry disease better known to the public. Patients with chronic kidney disease (CKD) who have no identified underlying cause should undergo Fabry disease testing according to this initiative. Fabry disease is a rare genetic condition that can cause kidney damage and other medical complications.
  • In May 2023, Chiesi Global Rare Diseases and Protalix BioTherapeutics announced that the U.S. Food and Drug Administration (FDA) had approved ELFABRIO (pegunigalsidase alfa-ix) in the United States for the treatment of adult patients with Fabry disease.

Fabry Disease Treatment Market Share, By Region, 2024 (%)

Asia Pacific is expected to host the fastest-growing Fabry disease treatment market over the forecast period. Several factors are driving the market's growth during the forecast period, including a surge in the geriatric population, the development of a broad range of Fabry disease treatments, and improved accessibility to pharmaceutical products. Moreover, the rise in inherited neurological disorders, the presence of critical generic pharmaceutical companies, and increased government initiatives and specialized communities positively impact market growth. Significant advancements and improvements in Fabry disease treatment in the region further contribute to this growth.

  • In November 2023, India’s Rajiv Gandhi Centre for Biotechnology announced that it would assist all districts, taluk hospitals, and family groups in Kerala to identify rare pediatric genetic diseases.

North America:

The North American Fabry disease treatment market is witnessing significant revenue growth owing to enhanced healthcare resources, timely and accurate diagnostics, and a strong flow of new treatments for the disease. The United States market is expected to dominate the region, with easier access to an array of treatment options, including enzyme replacement therapies and oral chaperone treatments.

The U.S. offers an advanced healthcare system that allows for accurate and timely treatment to be identified, in addition to sufficient reimbursement regulations for Fabry disease management, and increased professional education and awareness about treatment options for Fabry disease, which can collectively increase management of the disease. 
As long as there is ongoing research development, improvement in diagnostic practices and processes, and an appropriate structure for funding and reimbursement of healthcare services for rare genetic diseases, North America will remain a "place to be" for the Fabry disease treatment market, and will ultimately contribute to further patient growth and improvements in patient experiences and outcomes in the market across its regions.

North America held the largest share of the Fabry disease treatment market in 2024.

Asia-Pacific:

The Asia Pacific market for Fabry disease treatment is advancing rapidly, with a multitude of reasons for this growth. Awareness is rising in the Asia Pacific region for health care professionals, allowing for improved early diagnosis, and treatment leading to improved outcomes. Advances in diagnosis have led to additional patients being treated and treated patients have improved outcomes.

The acceleration of government support for rare disease programs and interest from biopharmaceutical companies committed to the area for Fabry disease treatment are driving this area in the Asia Pacific. Countries such as China, Japan, India, and South Korea are leading these developments in Fabry disease treatment, largely responsible for improving outcomes for people living with this rare disease due to higher levels of disposable income, accelerating urbanization, and an improved desire for more sophisticated health care. As a result, the Asia Pacific market should become a significant contributor of the global market for Fabry disease treatment.

Asia Pacific is projected to host the fastest growing market in the coming years.

Europe:

The Europe Fabry disease treatment market is growing steadily in all aspects. What drives this market is several factors. There is a huge well-developed healthcare system that allows for early diagnosis of Fabry disease and successful management. Some European countries have better environments overall, such as Germany, the UK, and France, as there are options for more advanced treatment as well as comprehensive healthcare for patients experiencing Fabry disease. 
More recently, with the availability of Enzyme Replacement Therapy's (ERT's) to oral chaperone therapies, that has increased options for patients and improved patient outcomes. Furthermore, there is strong reimbursement in place and readable policy developments as functional legislation to ensure the treatment of patients with Fabry disease is improving.

Added to the large scale introduction by healthcare providers in researching new developments and in development and more recently due to the covid-19 pandemic, there is more in depth research on options - Currently in Europe the level research and development continues to seem to enhance every type of therapeutic option available with promising new advancements in gene therapies and substrate reduction therapy's., thus Europe will always be a prominent place in the treatment of Fabry disease.

Market Overview

The Fabry disease treatment market is experiencing robust growth from increased usage of biotechnology, a wider understanding, and improved diagnostics capabilities. Fabry disease is a rare genetic disorder the condition is due to deficiency of the enzyme alpha-galactosidase A that requires lifelong management of the disease therefore treatment is essential.

The market has driven primarily by increased demand for ERTs such as agalsidase beta and oral chaperone therapies, such as migalastat. Further, as newer gene therapies now start to emerge, there is a shift in the treatment paradigm towards long-lasting or potentially curative treatment. There is a significant investment into research and development and partnerships between firms in biotechnology are creating faster pipelines. Expect continued growth in this market segment as patient advocacy leads to better genetic testing and knowledge of the disease.

Technological Advancement

Better patient outcomes result from recent therapeutic breakthroughs and newly developed diagnostic tools that enhance the diagnosis and management of this rare inherited metabolic disorder. The patient-specific approaches used in these therapies lead to improved treatment impacts and better quality of life. Modern gene therapies display considerable promise as a long-lasting cure for Fabry disease.

Fabry Disease Treatment Market Growth Factors

  • Rising demand for early-stage diagnosis
  • Increasing healthcare awareness
  • Innovation in Fabry disease treatment therapies
  • Increasing cases of Fabry disease across the globe

Market Scope

Report Coverage Details
Market Size in 2025 USD 2.45 Billion
Market Size in 2024 USD 2.27 Billion
Market Size by 2034 USD 4.87 Billion
Fabry Disease Treatment Market Growth Rate CAGR of 7.93% from 2025 to 2034
Largest Market North America
Base Year 2024
Forecast Period 2025 to 2034
Regions Covered North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa

Market Dynamics

Driver

The increasing prevalence of Fabry disease

The global fabry disease treatment market is expected to grow due to the increasing prevalence of the condition. Research indicates that Fabry disease affects 1 in 1,000 to 9,000 people. As the population grows, the likelihood of this hereditary disorder being passed on to newborns also rises. It has been observed that milder symptoms are more familiar with age than the severe form.

Fabry disease can affect individuals of all ethnicities and genders, leading to a broader patient base. Moreover, growing awareness of Fabry disease and increased discussions in the scientific and medical communities have led to better understanding and earlier diagnosis of the condition, further driving the Fabry disease treatment market growth.

  • In April 2024, Russia decided to launch the production of the Fabry disease drug this year. The project will be implemented by the Russian drugmaker Petrovax together with the Gamaleya Research Institute of Epidemiology and Microbiology, one of Russia’s leading medical and research institutions.

Restraint

Commodity price volatility and supply chain disruptions

Changes in the prices of raw materials, such as certain commodities, can affect the profit margins and operational costs of companies in the Fabry disease treatment market, exposing them to financial risks. Market saturation could also occur as similar products or services increase competition and put pressure on pricing and profitability, especially for companies with undifferentiated offerings.

Additionally, supply chain disruptions caused by natural disasters, geopolitical tensions, or other factors can hinder production and distribution, leading to delays, increased costs, and potential loss of market share in the Fabry disease treatment market.

Opportunity

Technology Advancements in the Fabry Disease Treatment Market:

The landscape of Fabry disease treatment is rapidly progressing due to advancements in technology which is influencing care and outcomes. First, next generation enzyme replacement therapies (ERTs) present the possibility of improved stability, shorter infusion times, as well as enhanced cellular uptake. 
Second, oral chaperone therapies (e.g. migalastat) provide a more convenient option for patients since they stabilize the misfolded enzyme. Third, gene therapies are emerging with the possibility of a durable, and potentially curative treatment, by treating the underlying genetic defect of Fabry disease. There are also changes in the factors due to enhanced diagnostic technologies, such as high throughput sequencing for genetics and biomarker measurement, which improve diagnosis speed and accuracy. 

Moreover, leveraging a deeper level of digital integration, it is becoming clearer how these advancements are improving the management of Fabry disease, treatment adherence, and the diagnostic journey. Collectively, these will help provide new treatments and personalized and evidenced-based care to Fabry disease.

Government Initiatives for Fabry Disease Treatment:

The treatment market for Fabry disease is benefiting from government action to stimulate research, development, and access for patients to therapies for rare diseases. Key drivers include orphan drug legislation incentives (market exclusivity, tax credits, fee waivers) for pharmaceutical companies and the availability of orphan drugs and potential treatments for Fabry Disease.

Regulatory authorities are allowing therapies to access fast-track and priority pathways that allow drugs to get to patients faster. Some governments are also funding clinical trials and natural history studies, absorbing the costs associated with early-stage research. Government action towards public-private partnerships and some national initiatives are also helping patients navigate the collaborative roads to diagnostics, screening and access to treatment. 
Other nations have launched rare disease frameworks. To assist with better access to effective therapies, some of these frameworks include dedicated funding, less stringent regulatory pathways to bring new treatments to market faster, or enhancements in health insurance funding and coverage for life-saving treatment options.

Government-wide mechanisms that are facilitating better innovation, collaboration, access, and ultimately, better outcomes for Fabry Disease patients are examples of the foundation that needs to be established to drive similar datasets in disease innovation and outcomes for rare diseases.

Distribution Channel Insights

The hospital pharmacy segment captured a significant portion of the market during 2024. 

  • In December 2024, the launch of Accelerate Pharmacy Solutions, a unified portfolio of solutions designed to improve support for hospital and health system customers optimize operations and improve was announced by Cencora, a global healthcare company.

Cencora Launches Accelerate Pharmacy Solutions to Enhance Support for Hospital and Health System Customers

The online pharmacy segment is projected to expand rapidly in the market in the coming years.

  • In August 2025, the launch of ‘Zepto Pharmacy’ an online pharmacy promising medicine delivery within 10 minutes in select metropolitan areas, was announced by Quick commerce platform Zepto.

Zepto launches online pharmacy, promises 10-minute medicine delivery - Check areas covered

Treatment Insights

The enzyme replacement therapy segment underwent notable growth in the market during 2024.

  • In September 2023, FDA approval and launch of new treatment for Pompe Disease was announced by Amicus Therapeutics. This component therapy is indicated for adults living with late-onset Pompe Disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

https://www.globenewswire.com/news-release/2023/09/28/2751407/15991/en/Amicus-Therapeutics-Announces-FDA-Approval-and-Launch-of-New-Treatment-for-Pompe-Disease.html

The substrate reduction therapy segment will gain a significant share of the market over the studied period of 2025 to 2034.

  • In July 2025, the launch of HARLIKU, the first and only FDA-approved treatment for reducing urinary homogentisic acid (HGA) in adult patients with alkaptonuria (AKU), was announced by Cycle Pharmaceuticals. AKU, also known as Black Bone Disease, is an ultra rare genetic metabolic disorder that affects approximately one in 1000000 in the U.S.

Cycle Pharmaceuticals launches HARLIKU™ (nitisinone) Tablets, the first FDA-approved treatment for use in Alkaptonuria (AKU)

Route of Administration Insights

The intravenous (IV) route of administration segment enjoyed a prominent position in the market during 2024. 

  • In December 2023, humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody ‘LEQEMBI® Intravenous Infusion’ (200mg, 500mg, lecanemab) launched in Japan was announced by Esai Co., Ltd. and Biogen Inc.

“LEQEMBI® Intravenous Infusion” (Lecanemab) for the Treatment of Alzheimer’s Disease to be Launched in Japan on December 20 | Biogen

The oral route of administration segment predicted to witness significant growth in the market over the forecast period. 

  • In May 2025, the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) submission for an investigational once daily, 25 mg oral formulation of Wegovy (Semaglutide) for chronic weight management in adults living with obesity or overweight and established cardiovascular disease was announced by Novo Nordisk.

FDA accepts filing application for oral semaglutide 25 mg, which if approved, would be the first oral GLP-1 treatment for obesity

Fabry Disease Treatment Market Companies

Recent Developments

  • In February 2025, uniQure completed enrolment in the first cohort of its Phase I/IIa clinical trial for AMT‑191, received a favorable IDMC safety recommendation, and began preparing for a second-dose cohort later in the first quarter. (Source- https://biotuesdays.com)
  • In February 2025 at the WORLD Symposium, Sangamo presented Phase 1/2 STAAR study results for isaralgagene civaparvovec (ST‑920) reporting positive kidney function trends and a strong safety profile, paving the way for accelerated approval discussions and planned BLA submission in 2026.  (Source- https://www.cgtlive.com)
  • In October 2024, the publication of results from the Phase-3 BRIGHT study of ELFABRIO (pegunigalsidase alfa-iwxj) 2 mg/kg administered every four weeks for 52 weeks in adult patients with Fabry disease who were previously treated with agalsidase alfa or beta administered every two weeks was announced by Chiesi Global Rare Diseases, a business unit of the Chiesi Group, established to deliver innovative therapies and solutions for people living with rare diseases. 
    Chiesi Global Rare Diseases Announces Publication of Results from Phase 3 BRIGHT Study of ELFABRIO® (pegunigalsidase alfa-iwxj) in Fabry Disease
  • In May 2024, an education and awareness campaign to increase visibility of Fabry Disease was launched by American Kidney Fund (AKF) in partnership with Sanofi Genzyme, a company working to support the rare disease community for more than 35 years.
    AKF and Sanofi Genzyme Launch Campaign for Awareness of Fabry Disease | Docwire News

Segments Covered in the Report

By Treatment

  • Enzyme Replacement Therapy (ERT)
  • Chaperone Treatment
  • Substrate Reduction Therapy (SRT)
  • Others

By Route of Administration

  • Intravenous
  • Oral

By Distribution Channel

  • Hospital Pharmacy
  • Online Pharmacy

By Geography

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • Middle East & Africa

For inquiries regarding discounts, bulk purchases, or customization requests, please contact us at sales@precedenceresearch.com

Frequently Asked Questions

The global fabry disease treatment market size is expected to increase USD 4.87 billion by 2034 from USD 2.27 billion in 2024.

The global fabry disease treatment market will register growth rate of 7.93% between 2025 and 2034.

The major players operating in the fabry disease treatment market are JCR Pharmaceuticals, Sanofi Genzyme, Green Cross Corporation, Chiesi Group, Regenxbio Inc., Protalix BioTherapeutics, Idorsia Pharmaceuticals, Amicus Therapeutics, and Others.

The driving factors of the fabry disease treatment market are the increasing prevalence of fabry disease and rising investments in healthcare departments.

North America region will lead the global fabry disease treatment market during the forecast period 2025 to 2034.

Ask For Sample

No cookie-cutter, only authentic analysis – take the 1st step to become a Precedence Research client

Meet the Team

Rohan Patil is one such expert who is helping shape the future of healthcare market insights with his deep industry knowledge and forward-thinking approach. Rohan holds a BSc. in Biotechnology and an MBA in Marketing, a combination that allows him to blend scientific understanding with strategic business acumen. With over 5 years of experience in the market research industry, Rohan has become a trusted analyst in the healthcare domain, where

Learn more about Rohan Patil

With over 14 years of experience, Aditi is the powerhouse responsible for reviewing every piece of data and content that passes through our research pipeline. She is not just an expert—she’s the linchpin that ensures the accuracy, relevance, and clarity of the insights we deliver. Aditi’s broad expertise spans multiple sectors, with a keen focus on ICT, automotive, and various other cross-domain industries.

Learn more about Aditi Shivarkar

Related Reports