There are currently 29 ongoing clinical trials involving Fabry Disease
Of the 29 trials,10 trials are in Phase IV
Furthermore, 8 trials are in Phase III
The global pharmaceutical industry is steadily developing new drugs for Fabry Disease, a genetic disorder. The largest number of ongoing clinical trials for Fabry Disease is conducted in North America. Europe and Asia-Pacific are among some of the other prominent regions engaged in Fabry Disease-related drug trials.
Protalix BioTherapeutics Inc: The leading ongoing Fabry Disease-related clinical trial sponsor
Protalix BioTherapeutics Inc is the top sponsor for Fabry Disease-related ongoing clinical trials.
Sanofi, Amicus Therapeutics Inc, Idorsia Pharmaceutical Ltd, Yonsei University, and Freeline Therapeutics Holdings Plc are among other notable clinical trial sponsors involved in Fabry Disease. A clinical trial sponsor can be a Company, Government, Individual, or Institution.
Marketed Drugs involving Fabry Disease
Agalsidase beta (Fabrazyme), Agalsidase alfa (Replagal), and Migalastat hydrochloride (Galafold) are key marketed drugs involving Fabry Disease.
Agalsidase beta (Fabrazyme) is a recombinant human a-galactosidase produced using recombinant (genetic) technology. It functions via Alpha Galactosidase A Replacement mechanism of action. It is formulated as a powder for solution for intravenous route of administration. Fabrazyme is indicated in adults, children, and adolescents aged 8 years and older for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency). Agalsidase beta was first approved in 2021 and is marketed globally including the US, the UK, Australia, Germany, China, and Japan by Genzyme Corp.
Agalsidase alfa (Replagal) is a human form of enzyme alpha-galactosidase A (a-Gal A). It functions via Alpha Galactosidase A Replacement mechanism of action. It is formulated as injectable, concentrate for a solution, for intravenous route of administration. Replagal is indicated to treat patients who have Fabry disease, a rare inherited disorder. The drug candidate was under development for the treatment of Fabry disease in the US. Agalsidase alfa was first approved in 2001 and is marketed globally including the UK, Australia, Germany, China, and Japan by Takeda Pharmaceuticals Australia Pty Ltd.
United States of America
China
Switzerland
United States of America
United States of America
United States of America
United States of America
Germany
France
Switzerland
Don’t wait - discover a universe of connected data & insights with your next search. Browse over 28M data points across 22 industries.
Access more premium companies when you subscribe to Explorer
Contact the team or request a demo to find out how our data can drive your business forward