SINGAPORE--JCR Pharmaceuticals has launched a Phase I clinical trial in Japan for JR-051, a recombinant alpha-galactosidase A, as an enzyme replacement therapy for Fabry's disease, the company said on Feb. 10.
The drug is a biosimilar version of Genzyme Japan's Fabrazyme (agalsidase beta) with a timeline of 2017 for regulatory approval.
The rare disease caused by the deficiency of the alpha-galactosidase A enzyme, is characterized by pain in the extremities, clouding of the cornea, renal failure and cardiac dysfunction. Roughly 300 patients in Japan and 5,000-10,000 worldwide suffer from the disease, according to JCR.
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