Fabry Disease Treatment Industry: Trends, Business Strategies and Opportunities 2025

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.

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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.