Development of a Biosimilar of Agalsidase Beta for the Treatment of Fabry Disease: Preclinical Evaluation

被引:0
作者
André B. P. van Kuilenburg
Carla E. M. Hollak
Ana Travella
Melisa Jacobs
Lucas D. Gentilini
René Leen
Karen M. M. Ghauharali-van der Vlugt
Femke S. Beers Stet
Susan M. I. Goorden
Sanne van der Veen
Marcelo Criscuolo
Mariana Papouchado
机构
[1] Amsterdam UMC location University of Amsterdam,Medicine for Society, Platform at Amsterdam
[2] Laboratory Genetic Metabolic Diseases F0-220,undefined
[3] Amsterdam Gastroenterology Endocrinology Metabolism,undefined
[4] Amsterdam UMC location University of Amsterdam,undefined
[5] Endocrinology and Metabolism,undefined
[6] UMC-University of Amsterdam,undefined
[7] Biosidus S.A.,undefined
来源
Drugs in R&D | 2023年 / 23卷
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页码:141 / 153
页数:12
相关论文
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[1]  
Schiffmann R(2015)Fabry disease Handb Clin Neurol 132 231-248
[2]  
Arends M(2017)Characterization of classical and nonclassical Fabry disease: A multicenter study J Am Soc Nephrol 28 1631-1641
[3]  
Wanner C(2019)Enzyme replacement therapy clears Gb3 deposits from a podocyte cell culture model of Fabry disease but fails to restore altered cellular signaling Cell Physiol Biochem 52 1139-1150
[4]  
Hughes D(2001)Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease N Engl J Med 345 9-16
[5]  
Mehta A(2002)Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy Kidney Int 62 1933-1946
[6]  
Oder D(2007)Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease J Am Soc Nephrol 18 1547-1557
[7]  
Watkinson OT(2017)Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: analysis of prognostic factors PLoS One 12 351-358
[8]  
Braun F(2018)Agalsidase alfa versus agalsidase beta for the treatment of Fabry disease: an international cohort study J Med Genet 55 162-168
[9]  
Blomberg L(2019)Antibodies against recombinant alpha-galactosidase A in Fabry disease: subclass analysis and impact on response to treatment Mol Genet Metab 126 2879-2889
[10]  
Brodesser S(2018)Dose-dependent effect of enzyme replacement therapy on neutralizing antidrug antibody titers and clinical outcome in patients with Fabry disease J Am Soc Nephrol 29 534-544