DelveInsight’s “Fabry Disease Market Insights, Epidemiology, and Market Forecast-2036″ report offers an in-depth understanding of the Fabry Disease, historical and forecasted epidemiology as well as the Fabry Disease market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
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Some of the key facts of the Fabry Disease Market Report:
The Fabry Disease market size was valued approximately USD 1,700 million in 2024 and is anticipated to grow with a significant CAGR during the study period (2022-2036).
In 2025, the United States accounted for the largest Fabry disease market, reaching approximately USD 920 million, surpassing the market sizes of the EU4 (Germany, Italy, France, and Spain), the United Kingdom, and Japan.
Among the EU4 (Germany, Italy, France, and Spain) and the UK, Germany recorded the largest Fabry disease market in 2025, valued at approximately USD 215 million, whereas Spain represented the smallest market, at nearly USD 85 million during the same year.
In Aug 2026, PTC Therapeutics secured the winning bid to acquire ST-920, a BLA-stage AAV gene therapy for Fabry disease, from Sangamo Therapeutics during a bankruptcy auction for $111 million upfront plus $100 million in potential milestones. ST-920 is a one-time treatment enabling long-term enzyme production. Supported by Phase 1/2 STAAR trial data showing sustained efficacy up to 4.5 years, PTC plans to complete the rolling BLA submission in Q4 2026 for a potential 2027 commercial launch.
In May 2026, Ongoing Clinical Development of ST-920 (Isaralgagene Civaparvovec), Sangamo Therapeutics continued advancing ST-920, one of the leading investigational gene therapies for Fabry disease. The program continued generating long-term follow-up data from the STAAR clinical study, with regulatory and development activities supporting its potential future commercialization.
In January 2026, Idorsia Pharmaceuticals presented Phase III MODIFY trial data showing a possible signal in patients with reduced baseline kidney function; however, no statistically significant renal benefit was observed with lucerastat in adults with Fabry disease. The regulatory pathway is expected to be discussed with the US FDA.In April 2026, Continued Advancement of Gene Therapy Programs, Multiple developers continued to advance liver-directed and AAV-based gene therapy candidates for Fabry disease, with ongoing evaluation of safety, enzyme expression, and biomarker improvements. Investigational programs remained focused on achieving durable α-Gal A production and reducing dependence on lifelong enzyme replacement therapy (ERT).
In March 2026, GT-GLA-S03 Received FDA Orphan Drug Designation, Glafabra Therapeutics announced that the FDA granted Orphan Drug Designation to GT-GLA-S03, an investigational autologous cell therapy for classic Fabry disease. The therapy is designed as a potentially redosable hematopoietic stem cell–based approach to provide continuous alpha-galactosidase A enzyme production and support long-term disease management.
In March 2026, Sangamo Therapeutics (Nasdaq: SGMO), a genomic medicine company, announced progress in its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration seeking accelerated approval for Isaralgagene civaparvovec (ST-920), an investigational gene therapy fully owned by the company and being developed for the treatment of adults with Fabry disease.
In May 2025, All patients treated with Sangamo Therapeutics’ gene therapy candidate ST-920 (isaralgagene civaparvovec) for Fabry disease in a Phase 1/2 trial have now reached the FDA’s required one-year milestone to proceed toward accelerated approval. According to Sangamo’s recent update, a pivotal data readout is anticipated by the end of June. The FDA previously indicated that data from the ongoing Phase 1/2 STAAR trial (NCT04046224) could be sufficient for accelerated approval, potentially eliminating the need for further clinical trials and speeding the therapy’s market entry.
In March 2025, Sangamo Therapeutics announced its agreement with the FDA on pursuing an accelerated approval pathway for ST-920 in Fabry disease, with a Biologics License Application (BLA) planned for submission in the latter half of 2025.
In February 2025, Isaralgagene civaparvovec (ST-920) is an experimental gene therapy designed to lower the abnormal buildup of specific fatty substances and potentially slow or stop the progression of Fabry disease. Developed by Sangamo Therapeutics, this therapy is intended to be administered through a single intravenous (IV) infusion. The U.S. Food and Drug Administration (FDA) has granted it Fast Track designation, Orphan Drug status, and Regenerative Medicine Advanced Therapy (RMAT) designation, recognizing its potential to address a serious unmet medical need.
In February 2025, Amicus Therapeutics shared updates on its migalastat development programs through oral presentations and poster sessions at the 21st Annual WORLD Symposium 2025.
ST-920, developed by Sangamo Therapeutics, is a promising gene therapy candidate for Fabry disease and is expected to receive U.S. approval by the second quarter (Q2) of 2026.
Among the emerging therapies for Fabry disease, Sanofi’s venglustat is one of the most advanced candidates, currently in the late stages of clinical development and anticipated to receive U.S. approval by 2027.
Based on DelveInsight’s assessment in 2025, the 7MM had approximately 18,000 diagnosed prevalent cases of Fabry disease.
In the 7MM, the US accounted for the highest number of diagnosed prevalent cases of Fabry disease, with nearly 9,400 cases in 2025.
In 2025, EU4 and the UK accounted for nearly 6,900 diagnosed prevalent cases of Fabry disease.
In 2025, there were ~700 and ~1,000 Fabry Disease diagnosed prevalent cases among males and females, respectively, in Japan.
In the US, the 10–19 years of age group had the highest number of cases, while the 40–49 years of age group accounted for the least number of cases.
Key Fabry Disease Companies: Chiesi and Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Protalix Biotherapeutics, AVROBIO, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, Greenovation Biotech GMBH, ICON plc, Chiesi Farmaceutici S.p.A., Amicus Therapeutics, Shire, and others
Key Fabry Disease Therapies: ELFABRIO (PRX-102), Venglustat, Isaralgagene civaparvovec (ST-920), PRX-102, AVR-RD-01, Venglustat, ST -920, FLT190, 4D-310, Lucerastat, Moss-aGal, PRX-102, pegunigalsidase alfa, migalastat, Agalsidase alfa, and others
The Fabry Disease epidemiology based on gender analyzed that Fabry Disease is more prevalent in males than females in the United States
The Fabry Disease market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Fabry Disease pipeline products will significantly revolutionize the Fabry Disease market dynamics.
Fabry Disease Overview
Fabry disease is a rare genetic disorder caused by mutations in the GLA gene, which leads to a deficiency or malfunction of the enzyme alpha-galactosidase A. This enzyme is crucial for breaking down a fatty substance called globotriaosylceramide (Gb3 or GL-3) in the body’s cells. When alpha-galactosidase A is deficient or dysfunctional, Gb3 accumulates in various tissues and organs, leading to a wide range of symptoms.
To Know in detail about the Fabry Disease market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Fabry Disease Market Forecast
Fabry Disease Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2022 to 2036. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Fabry Disease Epidemiology Segmentation:
The Fabry Disease market report proffers epidemiological analysis for the study period 2022–2036 in the 7MM segmented into:
Total Fabry Disease Diagnosed Prevalent Cases
Fabry Disease Gender-specific Diagnosed Prevalent Cases
Fabry Disease Age-specific Diagnosed Prevalent Cases
Fabry Disease Phenotype -specific Diagnosed Prevalent Cases
Download the report to understand which factors are driving Fabry Disease epidemiology trends @ Fabry Disease Epidemiology Forecast
Fabry Disease Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Fabry Disease market or expected to get launched during the study period. The analysis covers Fabry Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Fabry Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Fabry Disease Therapies and Key Companies
GALAFOLD (migalastat): Amicus Therapeutics
ELFABRIO (PRX-102/pegunigalsidase alfa): CHIESI Farmaceutici and Protalix Biotherapeutics
Venglustat: Sanofi (Genzyme)
Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics
PRX-102: Protalix Biotherapeutics
AVR-RD-01: AVROBIO
Venglustat: Sanofi Genzyme
ST-920: Sangamo Therapeutics
FLT190: Freeline Therapeutics
4D-310: 4D Molecular Therapeutics
Lucerastat: Idorsia Pharmaceuticals
Moss-aGal: Greenovation Biotech GMBH
PRX-102: ICON plc
pegunigalsidase alfa: Chiesi Farmaceutici S.p.A.
migalastat: Amicus Therapeutics
Agalsidase alfa: Shire
Discover more about therapies set to grab major Fabry Disease market share @ Fabry Disease Treatment Market
Fabry Disease Market Drivers
Advancements in Targeted Therapies: Continuous innovation in enzyme replacement therapies (ERTs), chaperone therapies, substrate reduction therapies, and gene therapies is expanding treatment options and driving market growth.
Increasing Awareness and Early Diagnosis: Improved newborn screening programs, genetic testing, and physician awareness are enabling earlier diagnosis, leading to timely treatment initiation and a larger diagnosed patient population.
Growing Investment in Rare Disease Research: Rising investments from pharmaceutical companies, government agencies, and orphan drug incentives are accelerating the development of novel Fabry disease therapies.
Robust Clinical Pipeline: An expanding pipeline of gene therapies, mRNA-based therapies, and next-generation ERTs is expected to address unmet needs and improve long-term patient outcomes.
Supportive Regulatory Environment: Orphan drug designations, priority review pathways, and other regulatory incentives are encouraging innovation and expediting the development of Fabry disease treatments.
Fabry Disease Market Barriers
Rare Disease with Limited Patient Population: The low prevalence of Fabry disease restricts the commercial potential of new therapies and makes patient recruitment for clinical trials challenging.
High Treatment Costs: Enzyme replacement therapies and emerging advanced therapies are expensive, creating affordability and reimbursement challenges in several healthcare systems.
Delayed and Missed Diagnosis: The disease often presents with nonspecific symptoms that overlap with other conditions, leading to delayed diagnosis and treatment initiation.
Lifelong Treatment Burden: Current standard-of-care therapies, particularly intravenous ERTs, require lifelong administration, impacting patient adherence and quality of life.
Clinical Development Challenges: Demonstrating long-term efficacy and safety, combined with small patient populations and disease heterogeneity, complicates clinical trials and regulatory approval processes.
Scope of the Fabry Disease Market Report
Study Period: 2022–2036
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Fabry Disease Therapies: ELFABRIO (PRX-102), Venglustat, Isaralgagene civaparvovec (ST-920), PRX-102, AVR-RD-01, Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, PRX-102, pegunigalsidase alfa, migalastat, Agalsidase alfa, and others
Fabry Disease Therapeutic Assessment: Fabry Disease current marketed and Fabry Disease emerging therapies
Fabry Disease Market Dynamics: Fabry Disease market drivers and Fabry Disease market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
Fabry Disease Unmet Needs, KOL’s views, Analyst’s views, Fabry Disease Market Access and Reimbursement
To know more about Fabry Disease companies working in the treatment market, visit @ Fabry Disease Clinical Trials and Therapeutic Assessment
Table of Contents
1. Fabry Disease Market Report Introduction
2. Executive Summary for Fabry Disease
3. SWOT analysis of Fabry Disease
4. Fabry Disease Patient Share (%) Overview at a Glance
5. Fabry Disease Market Overview at a Glance
6. Fabry Disease Disease Background and Overview
7. Fabry Disease Epidemiology and Patient Population
8. Country-Specific Patient Population of Fabry Disease
9. Fabry Disease Current Treatment and Medical Practices
10. Fabry Disease Unmet Needs
11. Fabry Disease Emerging Therapies
12. Fabry Disease Market Outlook
13. Country-Wise Fabry Disease Market Analysis (2022–2036)
14. Fabry Disease Market Access and Reimbursement of Therapies
15. Fabry Disease Market Drivers
16. Fabry Disease Market Barriers
17. Fabry Disease Appendix
18. Fabry Disease Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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