Global Fabry Disease Treatment Market Size Likely to Expand at a CAGR of 9.8% By 2034

Fabry Disease Treatment Market Size

As per the current market research conducted by the CMI Team, the Global Fabry Disease Treatment Market size is expected to record a CAGR of 9.8% from 2025 to 2034. In 2025, the market size is projected to reach a valuation of USD 3.10 Billion. By 2034, the valuation is anticipated to reach USD 7.02 Billion.

Fabry Disease Treatment Market Overview

A Rare inherited genetic condition classed as lysosomal storage disorder, Fabry disease—also known as Anderson-Fabry disease—is caused by mutations in the GLA gene, alpha-galactosidase A (α-GAL A) suffers either a deficit or malfunction.

Normally removed from cells, this enzyme breaks down a particular fatty molecule known as globotriaosylceramide (Gb3, GL-3). Particularly in the blood vessels, kidneys, heart, skin, and nervous system, the absence of functioning α-GAL A causes the gradual buildup of Gb3 in many cells and tissues throughout the body in Fabry disease. This accumulation can cause a wide spectrum of symptoms and disrupt proper cellular operation.

Fabry Disease Treatment Market Growth Factors and Dynamics

FDA Clearance Of Fabry Disease Drugs

The increasing FDA clearance for Fabry disease drugs is a major factor propelling the industry expansion over the analysis period. For instance, in September 2024, the U.S. Food and Drug Administration (FDA) granted GC Biopharma’s joint Fabry treatment project, “LA-GLA” (GC1134A/HM15421), with Hanmi Pharmaceutical, Investigational New Drug (IND) clearance for a Phase 1/2 clinical trial.

Co-developed by GC Biopharma and Hanmi Pharmaceutical, LA-GLA is a novel medication for the treatment of Fabry disease that is the first once-monthly subcutaneous treatment in history. The purpose of this clinical trial is to assess LA-GLA’s pharmacokinetics, pharmacodynamics, safety, and tolerability in Fabry disease patients.

Rising R&D Investment

The increasing investment and innovation in research and development (R&D) of novel and effective therapies for Fabry disease is creating an opportunity for the Fabry disease treatment market because it can help address patients’ unmet needs and challenges, such as high costs, limited availability, and variable efficacy of current treatments.

For example, gene therapy, stem cell treatment, and gene editing are new ways to repair genetic abnormalities or restore enzyme production in Fabry disease patients. These medicines have the potential to provide a cure or long-term treatment for Fabry’s disease.

Lack Of Awareness

In many respects, inadequate understanding and delayed diagnosis affect the market for treatments for Fabry disease. Because Fabry disease is rare and has unclear symptoms, doctors may not immediately recognize it and delay diagnosis.

Delaying the starting of required therapies affects patient outcomes and reduces demand for current medications. Moreover, since many of the people with Fabry disease go unnoticed, fewer of them get therapy. This underdiagnosis directly influences the market scale for treatments for Fabry disease.

Report Scope

Fabry Disease Treatment Market Recent Developments 

• In February 2024, mAbxience announced a major deal with Biosidus, a well-known Argentine biotechnology company. This CDMO (Contract Development and Manufacturing Organization) deal means that mAbxience will manufacture the active ingredient Agalsidase Beta, which Biosidus developed as a biosimilar to Fabrazyme.

List of the prominent players in the Fabry Disease Treatment Market:

• Sanofi S.A.
• Shire PIc.
• Amicus Therapeutics Inc.
• ISU Abxis Co Ltd.
• JCR Pharmaceuticals Co Ltd.
• Protalix Biotherapeutics Inc.
• Idorsia Pharmaceuticals Ltd.
• Avrobio Inc.
• Takeda Pharmkceutical Co Ltd.
• Chiesi Farmaceutici SpA
• Freeline Therapeutics Holdings PLC
• Yuhan Corp
• MOP Therapeutics
• Others

The Fabry Disease Treatment Market is segmented as follows:

By Treatment

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

By Route of Administration

• Intravenous Route
• Oral Route

By Distribution Channel

• Hospital Pharmacy
• Retail Pharmacy
• Online Pharmacy

Regional Coverage:

North America

• U.S.
• Canada
• Mexico
• Rest of North America

Europe

• Germany
• France
• U.K.
• Russia
• Italy
• Spain
• Netherlands
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• New Zealand
• Australia
• South Korea
• Taiwan
• Rest of Asia Pacific

The Middle East & Africa

• Saudi Arabia
• UAE
• Egypt
• Kuwait
• South Africa
• Rest of the Middle East & Africa

Latin America

• Brazil
• Argentina
• Rest of Latin America