Safety and effectiveness of enzyme replacement therapy with agalsidase alfa in patients with Fabry disease: Post-marketing surveillance in Japan

Molecular Genetics and Metabolism
Hiroaki SasaKoichi Kino

Abstract

Fabry disease is a rare X-linked inherited multisystem disorder resulting from deficiency of the lysosomal enzyme alpha-galactosidase A. Currently, specific therapies, including enzyme replacement therapies, are available for Fabry disease, but clinical trials provide limited information on long-term safety and effectiveness. Agalsidase alfa was approved in Japan in 2006. The post-marketing surveillance study of all patients receiving agalsidase alfa to evaluate its long-term safety and effectiveness as a mandatory condition for its approval had been conducted for 8 years (from February 2007 to March 2015). A total of 493 patients were included in this analysis of safety and effectiveness. The overall mean follow-up period was 3.5 years (range, 0.0-7.9 years). The percentage of patients with adverse drug reactions was 24.5% (121/493) and 12.6% had infusion-related reactions (62/493). In the 256 patients without prior enzyme replacement therapy whose IgG antibody data were available, 17 were IgG antibody positive (6.6%). However, the chronological correlation between seroconversion and the incidence of infusion-related reactions was not clear. The mean brief pain inventory score of the worst pain decreased in patients with moder...Continue Reading

Citations

Jul 1, 2020·Expert Opinion on Biological Therapy·Saptashwa DattaHatem Zayed
Aug 28, 2020·Orphanet Journal of Rare Diseases·Shinichiro YoshidaKimitoshi Nakamura
Oct 6, 2020·Molecular Genetics & Genomic Medicine·Takaaki SawadaKimitoshi Nakamura
Dec 2, 2020·Pain·Anthony J Burand, Cheryl L Stucky
May 18, 2021·Clinical Genetics·Vanessa CastelliClaudio Ferri

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