Fabrazyme Biosimilar Marketed in Japan Under New Agreement

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by Vanda Pinto, PhD |

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JCR Pharmaceuticals and Sumitomo Dainippon Pharma have teamed up to market and commercialize in Japan agalsidase beta BS, a biosimilar of Fabrazyme, an already approved treatment designed to replace a critical enzyme missing in people with Fabry disease.

According to a company press release, “This marketing alliance between the two companies will further enhance both partner’s contribution to the treatment of Fabry disease patients.”

In Fabry disease, the alpha-galactosidase A (Gal A) enzyme is missing or dysfunctional because of mutations in the GLA gene. Not enough Gal A enzyme results in the toxic buildup of two fatty substances — Gb3 and lyso-Gb3 — in tissues and organs such as the kidneys, heart, blood, and skin. Over time, this buildup can lead to irreversible damage and serious complications.

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Fabrazyme (agalsidase beta), developed by Sanofi Genzyme, and Replagal (agalsidase alfa), developed by Shire, are two enzyme replacement therapies (ERT) that deliver a lab-produced Gal A copy, effectively generating the enzyme that is nonfunctional or missing.

Agalsidase beta BS, also known as JR-051, is a biosimilar of Fabrazyme developed in Japan by JCR Pharmaceuticals in collaboration with Amicus Therapeutics and GlaxoSmithKline. A biosimilar is a biological product that is almost identical to an already approved treatment in regard to quality, effectiveness, and safety.

Preclinical studies in cultured cells, rats, nonhuman primates, and a mouse model of Fabry disease showed that JR-051 and Fabrazyme had comparable pharmacokinetic properties, meaning that they are distributed in the body, metabolized, and excreted in a similar way.

In mice, JR-051 was found to be just as effective as Fabrazyme at reducing Gb3 levels in several organs, and its safety was also proved in nonhuman primates.

JR-051’s safety and efficiency has also been demonstrated in Phase 1 and 2/3 clinical trials. The results demonstrated that JR-051 had a similar safety profile as Fabrazyme and was effective at reducing Gb3 and lysoGb3 blood levels.

JR-051 has been sold in Japan as agalsidase beta BS since November 2018. The agreement lets Sumitomo Dainippon Pharma exclusively market and distribute the therapy and deliver information about it to healthcare professionals, starting April 1.