Fabry Disease Treatment Market

The global Fabry disease treatment market size was USD 2.16 billion in 2022 and is projected to reach USD 4.81 billion by 2031, expanding at a CAGR of 9.3 % during the forecast period, 2023–2031. The growth of the market is attributed to increasing incidence of the disease coupled with increasing adoption of novel therapies such, as chaperone treatment.

Fabry disease is an X-linked lysosomal storage illness that causes progressive organ failure due to lack of the alpha-galactosidase enzyme. An abnormal buildup of a specific fatty substance named globotriaosylceramide in numerous human tissues, including eyes, skin, kidneys, gastrointestinal tract, brain, heart, and central nervous system, causes this condition.
 

Patients with Fabry disease can have impaired kidney function that can lead to full renal failure, early heart condition, and disabling gastrointestinal symptoms. As there is no cure for Fabry disease, treatment focuses on preventing complications and alleviating symptoms. Enzyme replacement therapy (ERT), chaperone therapy, and substrate reduction therapy are some of the current treatments.

There were no viable treatments for Fabry before enzyme replacement therapy, and the typical life expectancy of a Fabry patient was up to 50 years old. The disease's late onset and mild symptoms leads to number of patients undiagnosed. Sanofi estimates that approximately 3,000 patients in the US suffer from Fabry disease Patients with Fabry disease live longer lives on average than patients with other lysosomal storage disorders.

Market Trends, Drivers, Restraints, and Opportunities

• Increasing R&D activities and the potential approval of promising pipeline drugs such as substrate reduction therapies and enzyme replacement therapies are expected to propel the market during the period.
• Rising strategic collaboration and licensing agreement between the companies is expected to fuel the market growth during the forecast period.
• The preference for substrate reduction therapies and gene therapies in the healthcare industry is expected to drive the growth of the industry players in the projected timeline.
• Growing emergence of drugs used in the treatment of the risk associated with Fabry disease is anticipated to spur the market growth during the forecast period.
• Effective treatment is either unavailable or unaffordable, which can hamper the market growth.
• Extensive R&D activities and potential approval of promising pipeline products such as substrate reduction therapies and enzyme replacement therapies are projected to fuel the market expansion in coming years.

Scope of Fabry Disease Treatment Market Report

The report on the global Fabry disease treatment market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.

Fabry Disease Market Segment Insights

Enzyme replacement therapy segment is projected to account a key market share

Based on treatments, the market is segmented into enzyme replacement therapy, chaperone treatment, substrate reduction therapy, and others. The enzyme replacement therapy (ERT) segment is expected to account for a key market share during the forecast period owing to strong sales of Fabrazyme and Replagal, as well as the potential approval of promising pipeline candidates.

However, the introduction of new effective therapeutic options, such as gene therapies can reduce the market share of the segment during the forecast period. ERT is the gold standard for illness management. Although Sanofi's Fabrazyme and Shire's Replagal have received European approval, only Fabrazyme has received approval in the US.

Clinical trials are focusing on improving the safety and efficacy profile of ERTs, as well as the development of innovative oral medicines can replace intravenous infusions. Galafold, a drug developed by Amicus Therapeutics, was recently licensed in the US, Canada, the European Union, Japan, Australia, Israel, and South Korea, as the first oral chaperone medication for adult patients.

Asia Pacific is anticipated to dominate the market

On the basis of regions, the Fabry disease treatment market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. Asia Pacific is anticipated to expand at an impressive CAGR during the forecast period. The regional market growth is attributed increasing healthcare expenditure and improving infrastructure. Rising population in emerging countries such as India and China is expected to propel the market growth in the region.

In 2020, North America accounted for a major market share, followed by Europe. Higher acceptance of new therapies, improved healthcare facilities, and advantageous reimbursement policies are contributing to the regional market growth. Drug companies are increasing their R&D efforts in the field of rare diseases as a result of improvement in health insurance schemes that are covering expensive treatments and favourable government healthcare regulations are driving the market growth in North America.

Segments

The global Fabry disease treatment market has been segmented on the basis of

Treatments

• Enzyme Replacement Therapy
• Chaperone Treatment
• Substrate Reduction Therapy
• Others

Regions

• Asia Pacific
• North America
• Latin America
• Europe
• Middle East & Africa

Key Players

• Sanofi S.A.
• Shire Plc.
• Amicus Therapeutics Inc.
• ISU Abxis Co Ltd.
• JCR Pharmaceuticals Co Ltd.
• Protalix Biotherapeutics Inc.
• Idorsia Pharmaceuticals Ltd.
• Avrobio Inc.
• Greenovation Biotech GmbH
• Moderna Therapeutics Inc.
• Green Cross Pharma Pte Ltd.

Competitive Landscape

Key players competing in the Fabry disease treatment market include Sanofi S.A., Shire Plc., Amicus Therapeutics Inc., ISU Abxis Co Ltd., JCR Pharmaceuticals Co Ltd., Protalix Biotherapeutics Inc., Idorsia Pharmaceuticals Ltd., Avrobio Inc., Greenovation Biotech GmbH, Moderna Therapeutics Inc., and Green Cross Pharma Pte Ltd.

Sanofi’s sales of medicine Fabrazyme is helping the company to account for a major market share. During the forecast period, Sanofi and Shire are expected to encounter significant competition from biosimilar releases.

Meanwhile, Amicus Therapeutics’s Galafold, the first oral treatment for Fabry disease, is aiding the company to become a major player in the rare disease space. On the other hand, Idorsia and Avrobio are expected to command a large share of the market with their drug lucerastat and AVR-RD-01, respectively.