Fabry Disease Market Set for Robust Growth by 2034, Driven by Gene Therapy Breakthroughs and Expanding Pipeline from Sangamo, Sanofi, Amicus & Emerging Innovators | DelveInsight

Fabry Disease Market

DelveInsight’s “Fabry Disease Market Insights, Epidemiology, and Market Forecast-2034″ 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 (2020-2034).
• 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.
• In December 2024, Exegenesis Bio, a global clinical-stage gene therapy company, announced that the U.S. FDA has granted Orphan Drug Designation (ODD) to EXG110, an innovative gene therapy for Fabry disease—a rare lysosomal disorder characterized by excessive lipid accumulation in tissues, leading to renal failure, cardiac complications, and strokes.
• In 2024, the United States held the largest share of the Fabry Disease Therapeutics Market among the 7MM, with an estimated value of around USD 880 million. This market is projected to grow at a notable compound annual growth rate (CAGR) over the forecast period from 2020 to 2034.
• In 2024, Germany recorded the largest Fabry Disease Treatment Market Size among European countries, reaching approximately USD 210 million. In contrast, Spain had the smallest market size, with an estimated value of around USD 82 million.
• In 2024, Japan’s Fabry Disease Therapeutics Market Size was estimated at approximately USD 150 million, representing around 9% of the overall market across the 7MM.
• The anticipated introduction of upcoming therapies like ST-920 is expected to drive changes in the overall Fabry Disease market size in the coming years.
• In 2024, the total number of diagnosed prevalent cases of Fabry Disease in the United States was estimated to be approximately 9,200.
• In 2024, the United States accounted for the largest share of the diagnosed prevalent population of Fabry Disease, representing approximately 52% of the total across the 7MM. Meanwhile, the combined share of EU4 and the UK made up around 38%, and Japan contributed roughly 10% of the total diagnosed population.
• In 2024, among the EU4 countries and the UK, Germany had the highest number of diagnosed prevalent Fabry Disease cases (around 2,170), followed by the UK, while Spain reported the lowest number of cases.
• In 2024, the estimated number of diagnosed prevalent cases in the US included approximately 3,300 individuals with the classic phenotype and around 5,860 with the late-onset phenotype.
• 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 2020 to 2034. 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 2020–2034 in the 7MM segmented into:

• Total Prevalence of Fabry Disease
• Prevalent Cases of Fabry Disease by severity
• Gender-specific Prevalence of Fabry Disease
• Diagnosed Cases of Episodic and Chronic Fabry Disease

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

Venglustat: Sanofi (Genzyme)

Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipids accumulation. The drug inhibits the enzymatic conversion of ceramide to glucosylceramide, reducing available substrate for the synthesis of more complex glycosphingolipids. It is getting evaluated in the Phase III for Fabry disease.

Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics

ST-920 is a liver-tropic rAAV 2/6 vector carrying the cDNA for human alpha-galactosidase-A that is delivered through a single dose IV infusion. The drug aims to deliver a working copy of the galactosidase-α gene to the liver so that liver cells can start producing functional alpha-galactosidase-A. ST-920, in the Phase I/II for Fabry disease, has the potential as a one-time, durable treatment option for Fabry disease that can improve patient outcomes.

Fabry Disease Therapies and Key Companies

• ELFABRIO (PRX-102): Chiesi 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 Strengths

• Emphasis on NBS to detect Fabry Disease at an early stage allows for timely treatment with better risk- benefit assessment.
• The growing Fabry Disease pool is expected to propel the treatment market, adding opportunities for new pharma players.

Fabry Disease Market Opportunities

• Development of potential biomarkers allows tracking the severity and predicting the progression of the disease, as well as checking the effectiveness of therapeutic intervention.
• The shortcomings of enzyme replacement therapy, partly due to antibody formation, can be alleviated by combining new oral therapy approaches.

Scope of the Fabry Disease Market Report

• Study Period: 2020–2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• 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
• 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 (2020–2034)

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

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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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