The global fabry disease treatment market size is estimated to reach a value of USD 3.12 billion by 2025, according to a new report by Grand View Research, Inc., exhibiting a CAGR of 9.6% during the forecast period. Rising cohort of Fabry disease, increasing adoption of novel therapies such as chaperone treatment, and potential approval of pipeline drugs including substrate reduction therapies and enzyme replacement therapies are providing a fillip to the market.
Fabry disease is a rare inherited lysosomal storage disorder caused by genetic mutation that interferes with function of alpha galactosidase enzyme. Fabry disease generally has a late onset with mild symptoms, due to which several patients are often undiagnosed. Standard of care for management of the disease is enzyme replacement therapy (ERT). Sanofi’s Fabrazyme and Shire’s Replagal are the only approved ERTs in the EU; however in the U.S., only Fabrazyme has been approved.
Focus of current clinical trials is to improve the safety and efficacy profile of ERTs and introduction of novel oral therapies that can eliminate the need for intravenous infusions. Recently, Amicus Therapeutics’ Galafold has been approved as the first oral chaperone therapy for the treatment of adults in the U.S., Canada, EU, Japan, Australia, Israel, and South Korea.
North America held the largest share in the Fabry disease treatment market in 2017, followed by Europe. The growth of the region can be attributed to higher adoption of novel therapies, favorable reimbursement policies, and better healthcare facilities. However, high cost of therapy with ERTs such as Fabrazyme is an impediment for the market in developing regions.
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Further Key Findings From the Study Suggest:
- Although the exact prevalence is unknown, Fabry disease affects approximately one in 40,000 males
- The enzyme replacement therapy segment led the market with over 85.0% market share in 2017 due to strong sales of Fabrazyme and Replagal.
- North America commanded the leading market share of more than 45.0% in the Fabry disease treatment market, followed by Europe, in 2017
- APAC is likely to witness the fastest growth during the forecast period, followed by Latin America, due to increasing healthcare expenditure and rising patient population
- Some of the key players in the market are 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.
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