Fabry Disease Treatment Market Report and Forecast 2025-2034

The Fabry disease treatment market was valued at USD 2.75 Billion in 2024 driven by innovations in treatment options across the 8 major markets. It is expected to grow at a CAGR of 9.90% during the forecast period of 2025-2034 and attain a market value of USD 7.07 Billion by 2034.

Fabry Disease Treatment Market Overview

Fabry disease is a rare, inherited lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase A. This enzyme is crucial for breaking down lipids in the body. Without it, harmful levels of lipids accumulate in cells, leading to severe health problems such as kidney failure, heart issues, strokes, and skin conditions, affecting various organs over time.

Fabry Disease Treatment Market Growth Drivers

Advent of Advanced Treatments and Genetic Therapy Innovation to Accelerate Market Expansion

The market is witnessing growth driven by advancements in gene therapy and the rising need for effective treatments for rare diseases. For instance, in December 2024, Exegenesis Bio received an orphan drug designation from the FDA for EXG110, a novel gene therapy to treat Fabry disease. This therapy works by delivering a genetic payload directly to liver and heart cells, offering a potentially more effective, safer, and easier one-time treatment. Exegenesis Bio's progress in China, along with plans for a US-based clinical trial, is expected to enhance the market's development, offering new hope to patients. The approval of EXG110 could boost market value, particularly in regions where alternative therapies are limited, driving growth in the forecast period.

Rising Focus on Rare Disease Treatments and FDA Approvals to Meet the Growing Fabry Disease Treatment Market Demand

The increasing focus on rare disease therapies and the successful development of innovative gene therapies propel the growth of the market. For instance, in September 2024, uniQure N.V. received an FDA orphan drug designation for its investigational gene therapy, AMT-191, targeting Fabry disease. AMT-191 is currently undergoing Phase I/IIa trials in the US, with the first patient having been dosed in August 2024. This development is poised to have a significant impact on the market by introducing a potential breakthrough treatment for Fabry disease. With a promising clinical trial underway, AMT-191 could open doors to new treatment avenues, potentially expanding the market and enhancing therapeutic options in the forecast period.

Fabry Disease Treatment Market Trends

The market is witnessing several trends and developments to improve the current scenario. Some of the notable trends are as follows:

Growing Adoption of Gene Therapies for Treatment

Gene therapies are gaining significant traction in the Fabry disease treatment market due to their potential to provide long-term or even curative solutions. These therapies focus on delivering functional copies of the missing enzyme, alpha-galactosidase A, offering a one-time treatment option. For instance, in February 2024, Sangomo Therapeutics received alignment from the FDA following a Type B interaction, advancing plans for accelerated approval of its investigational gene therapy, isaralgagene civaparvove (ST-920), for Fabry disease. Based on promising data from the ongoing STAAR trial, Sangamo aims to submit a biologics license application (BLA) in late 2024. As gene therapies advance, they are expected to reduce the reliance on enzyme replacement therapies, boosting market growth and development during the forecast period.

Enzyme Replacement Therapy Innovations to Enhance Fabry Disease Treatment Market Value

Enzyme replacement therapy (ERT) remains a primary treatment for Fabry disease, with several new formulations entering the market. These advanced ERTs aim to improve patient outcomes with better dosing schedules and fewer side effects. With the growing patient population and continued innovation in ERT formulations, the market value for Fabry disease treatments is projected to rise, leading to greater patient access and treatment efficacy.

Rising Investments to Meet Fabry Disease Treatment Market Demand

The increasing investment in rare disease treatments is a key trend shaping the Fabry disease treatment market. Pharmaceutical companies are dedicating more resources to developing effective therapies for rare genetic disorders like Fabry disease, driven by orphan drug incentives and regulatory support. This influx of investment is expected to foster rapid market expansion, enabling faster advancements in treatment options and improving accessibility for patients.

Adoption of Personalised Medicine to Shape Fabry Disease Treatment Market Landscape

Personalised medicine is becoming a growing trend in the treatment of Fabry disease, with therapies tailored to individual genetic profiles and disease progression. These treatments, including gene therapies and enzyme therapies, aim to provide more targeted and effective results. As precision medicine continues to evolve, the Fabry disease treatment market is expected to experience increased demand, with a shift towards more customised, patient-centric care.

Fabry Disease Treatment Market Segmentation

The market report offers a detailed analysis of the market based on the following segments:

Market Breakup by Treatment

• Enzyme Replacement Therapy
• Oral Chaperone Therapy
• Others

Market Breakup by Route of Administration

• Oral Route
• Intravenous Route

Market Breakup by Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies

Market Breakup by Region

• United States
• United Kingdom
• Germany
• France
• Italy
• Spain
• Japan
• India

Fabry Disease Treatment Market Share

Enzyme Replacement Therapy to Lead the Market Share by Treatment

Enzyme Replacement Therapy (ERT) is expected to hold the largest share in the Fabry disease treatment market due to its established efficacy in managing the disease. ERT provides long-term symptom relief by replacing the deficient enzyme, alpha-galactosidase A. Its widespread adoption, along with proven clinical success, continues to drive significant market demand compared to other treatment options.

Fabry Disease Treatment Market Analysis by Region

Regionally, the market report offers insights into the United States, United Kingdom, Germany, France, Italy, Spain, Japan and India. Among these, the United States holds the largest market share due to its well-established healthcare infrastructure, high treatment adoption rates, and significant investment in rare disease therapies. Additionally, the presence of leading pharmaceutical companies and robust regulatory support drive the demand for advanced treatments in the country.

Leading Players in the Fabry Disease Treatment Market

The key features of the market report comprise patent analysis, clinical trials analysis, grants analysis, funding and investment analysis, and strategic initiatives by the leading players. The major companies in the market are as follows:

Sanofi SA

Headquartered in Paris, France, Sanofi SA was established in 2004. The company is a global leader in healthcare, with a strong presence in the Fabry disease treatment market. Sanofi’s portfolio includes enzyme replacement therapies such as Fabrazyme, which is approved for the treatment of Fabry disease, addressing unmet medical needs with innovative therapies for rare genetic conditions. In February 2023, Sanofi Speciality Care reaffirmed its commitment to improving the lives of patients with rare diseases in India. Over the past 40 years, the company has launched therapies for conditions like Gaucher, Fabry, Pompe, Mucopolysaccharidosis I, and Acid Sphingomyelinase deficiency (ASMD). Sanofi is also set to introduce two new therapies, Nexviazyme and Xenpozyme, for Pompe and Niemann-Pick disease, following orphan drug status and approval in several countries. These efforts highlight Sanofi's leadership in the rare disease treatment space.

M6P Therapeutics Inc

M6P Therapeutics Inc., founded in 2020, is a biotechnology company based in the United States. The company is focused on developing gene therapies for rare genetic diseases, including Fabry disease. M6P's flagship product aims to correct the underlying genetic defect in Fabry patients, offering a potential one-time treatment that could revolutionise current approaches to disease management.

Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited, founded in 1781 and headquartered in Osaka, Japan, is a global biopharmaceutical leader. In the Fabry disease treatment space, Takeda offers Replagal, an enzyme replacement therapy designed to replace the deficient alpha-galactosidase A enzyme in patients with Fabry disease. The company is dedicated to addressing rare and complex diseases with advanced, patient-centric therapies. In March 2024, Takeda extended its collaboration with Centogene to enhance diagnostic services for lysosomal storage disorders, including Fabry disease. The partnership, initially launched to improve access to fast and reliable diagnostics for rare diseases like Gaucher and Hunter syndrome, will continue to support patients in receiving accurate diagnoses. This extended collaboration strengthens Takeda’s commitment to improving patient outcomes and advancing the treatment landscape for lysosomal storage disorders, driving innovation in the rare disease space.

Yuhan Corporation

Founded in 1926, Yuhan Corporation is a South Korean pharmaceutical company headquartered in Seoul. The company has been expanding its portfolio to include treatments for rare diseases like Fabry disease. Yuhan collaborates with global partners to develop innovative therapies, including enzyme replacement treatments aimed at improving outcomes for patients suffering from Fabry disease and other lysosomal storage disorders.

Other key players in the market include Freeline Therapeutics Holdings PLC, Chiesi Farmaceutici S.p.A., Protalix BioTherapeutics Inc., JCR Pharmaceuticals Co., Ltd, Amicus Therapeutics, Inc., and ISU ABXIS.

Key Questions Answered in the Fabry Disease Treatment Market

• What was the Fabry disease treatment market value in 2024?
• What is the Fabry disease treatment market forecast outlook for 2025-2034?
• What is the market breakup based on the treatment?
• What is the market breakup based on the route of administration?
• What is the market breakup based on the distribution channel?
• How has the market performed so far and how is it anticipated to perform in the coming years?
• What are the market's major drivers, opportunities, and restraints?
• Which country is expected to experience expedited growth during the forecast period?
• What are the major Fabry disease treatment market trends?
• Which treatment will lead the market segment?
• Which route of administration will lead the market segment?
• Which distribution channel will lead the market segment?
• Who are the key players involved in the Fabry disease treatment market?
• What is the patent landscape of the market?
• What are the current unmet needs and challenges in the market?
• How are partnerships, collaborations, mergers, and acquisitions among the key market players shaping the market dynamics?

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