Agalsidase Beta BS I.V. Infusion [JCR] (JR-051) for Fabry Disease: Notice on the Publication of the Results of the Phase 1 and 2/3 Clinical Trials in Molecular Genetics and Metabolism

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that the results of the phase 1 and 2/3 clinical trials of Agalsidase Beta BS I.V. Infusion [JCR] (JR-051), recombinant Agalsidase Beta, for Fabry disease have been published in the electronic edition of Molecular Genetics and Metabolism, the official journal of Society for Inherited Metabolic Disorders. This is JCR’s first product for enzyme replacement therapy (ERT) for Lysosomal Storage Disorders (LSDs), also the first of the kind manufactured in Japan. Agalsidase Beta BS I.V. Infusion [JCR] has been launched since November 2018 as the first biosimilar for the treatment of rare diseases. A summary of the article is as follows.

 Title: Pharmacokinetics and pharmacodynamics of JR-051, a biosimilar of agalsidase beta, in healthy adults and patients with Fabry disease: Phase I and II/III clinical studies

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 Summary The Phase 1 and 2/3 studies were conducted with the aim to verify clinical comparability of JR051 and an upfront biopharmaceutical (agalsidase beta). The results demonstrated that JR051 and agalsidase beta are comparable in terms of efficacy and safety.

【Phase 1 study】 20 healthy adult male volunteers were administered JR-051 and agalsidase beta to confirm pharmacokinetic equivalence in a randomized, double-blind, parallel-group manner. The study demonstrated comparable pharmacokinetic profiles of JR-051 and agalsidase beta.

【Phase 2/3 study】 16 patients with Fabry disease underwent treatment with agalsidase beta (1mg/kg, once every other week), then were switched to intravenous administrations of JR-051 (1 mg/kg, once every other week).

・Efficacy:The 95% confidence intervals of the ratios of the GL-3 plasma concentrations (primary endpoint) during the agalsidase beta treatment, as well as those of Lyso-GL-3, to the respective plasma concentrations after 26 and 52 week-administrations of JR-051 were within pre-determined equivalence acceptability ranges.

・Safety:No severe infusion associated reactions (IARs), such as anaphylactic shock, were observed. One IAR, commonly observed with the ERT for Fabry disease, was reported in a patient after JR-051 administration.

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