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Emerging Trends Influencing The Growth Of The Fabry Disease Market: Reducing Immunogenic Responses In Enzyme Replacement Therapy

06-11-2025 11:48 AM CET | Health & Medicine

Press release from: The Business Research Company

Fabry Disease

Fabry Disease

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How Big Is the Fabry Disease Market Size Expected to Be by 2034?
The market size for Fabry disease has demonstrated robust growth in the recent past, set to advance from $2.10 billion in 2024 to $2.28 billion in 2025, corresponding to a compound annual growth rate (CAGR) of 8.2%. Multiple factors contributed to this growth during the historical period, including heightened awareness and identification of rare diseases, expansion in specialized healTH*Care infrastructure, government incentive programs, orphan drug designations, rising investment in research for rare diseases, and initial rollouts of therapies.

The market for Fabry disease treatments is set to experience substantial growth in the coming years, reaching a worth of $3.10 billion in 2029 - a compound annual growth rate (CAGR) of 8.0%. This growth during the forecast period can be accredited to numerous factors, including the development of newborn screening programs, regulatory backing for drugs treating orphan and rare ailments, enhanced access to healTH*Care, the rise of telemedicine and remote care, and an increasing demand for tailored medicine. Key trends for the forecast period encompass the uptake of enzyme replacement therapy, improvements in genetic testing, utilization of AI and digital tools for early diagnosis, AI-based diagnostic algorithms, and the progression of gene therapy.

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What Are the Emerging Segments Within the Fabry Disease Market?
The fabry disease market covered in this report is segmented -

1) By Type: Type 1, Type 2, Other Types
2) By Treatment: Enzyme Replacement Therapy, Oral Therapy, Adjunct Therapy, Other Treatments
3) By Diagnosis: Blood Test, Genetic Test, Parenteral Test, Other Diagnosis
4) By Distribution Channel: Hospital Pharmacy, Online Pharmacy, Retail Pharmacy
5) By End User: Hospitals, Homecare, Specialty Clinics, Other End Users

Subsegment:

1) By Type 1: Classic Fabry Disease, Later-Onset Fabry Disease
2) By Type 2: Cardiovascular-Focused Fabry Disease, Renal-Focused Fabry Disease
3) By Other Types: Variant Fabry Disease, X-linked Fabry Disease

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What Long-Term Drivers Are Shaping Fabry Disease Market Trends?
The fabry disease market is projected to expand due to an increasing emphasis on personalized medicine. This treatment strategy uses data about an individual's genetics, environment, and lifestyle to create therapies specifically designed for them, resulting in improved results. The attraction towards personalized medicine is due to its potential to enhance treatment efficiency and decrease side effects by making therapies specific to individual patient needs. It helps formulate treatment strategies based on the distinct genetic mutation and disease intensity of a patient, optimizing results and curtailing unneeded side effects. For example, the Personalized Medicine Coalition, a non-profit based in the U.S., in February 2024, in 2023, the FDA issued approval for 16 new personalized treatments for patients with rare diseases, a significant rise from the six approvals in 2022. As a result, the fabry disease market is benefiting from the increasing focus on personalized medicine.

Who Are the Top Competitors in Key Fabry Disease Market Segments?
Major companies operating in the fabry disease market are Sanofi S.A., GlaxoSmithKline plc, Takeda Pharmaceutical Company Limited, JCR Pharmaceuticals Co. Ltd., Sumitomo Pharma Co. Ltd, Chiesi Farmaceutici S.p.A., Amicus Therapeutics Inc., Idorsia Pharmaceuticals Ltd, Sangamo Therapeutics Inc., uniQure N.V., Protalix BioTherapeutics Inc., Spur Therapeutics Inc., Exegenesis Bio Inc., ISU ABXIS Co. Ltd., Eleva GmbH, AceLink Therapeutics Inc., 4D Molecular Therapeutics Inc., iBio Inc., Freeline Therapeutics Holdings plc, Greenovation Biotech GmbH

What Are the Latest Developing Trends in the Fabry Disease Market?
Key players in the Fabry disease market are directing their efforts towards the development of novel therapies such as PEGylated enzyme replacement therapy (ERT). These innovative therapies aim to heighten treatment effectiveness, increase patient adherence, and limit the immune responses typically observed with standard ERTs. PEGylated ERT is a sophisticated version of the regular ERT, with polyethylene glycol (PEG) chains linked to the enzyme, thus enhancing its stability, extending its presence in the body, and mitigating any immune response. For example, in May 2023, Chiesi Global Rare Diseases, a subsidiary of Italy-based Chiesi Group, got the nod from the U.S. Food and Drug Administration (FDA) for PRX-102 (pegunigalsidase alfa) intended to treat adult Fabry disease patients. This approval is backed by an intensive clinical program undertaken with over 140 patients with long-term monitoring of up to 7.5 years, showcasing the treatment's ability to decrease renal Gb3 deposits and uphold kidney function, all the while being safe.

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Which Regions Are Becoming Hubs for Fabry Disease Market Innovation?
North America was the largest region in the fabry disease market in 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the fabry disease market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

Frequently Asked Questions:
1. What Is the Market Size and Growth Rate of the Fabry Disease Market?
2. What is the CAGR expected in the Fabry Disease Market?
3. What Are the Key Innovations Transforming the Fabry Disease Industry?
4. Which Region Is Leading the Fabry Disease Market?

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