Agalsidase-Beta Therapy for Advanced Fabry Disease

Abstract

Agalsidase-beta therapy slowed progression to the composite clinical outcome of renal, cardiac, and cerebrovascular complications and death compared with placebo in patients with advanced Fabry disease. Therapeutic intervention before irreversible organ damage may provide greater clinical benefit.

Effects of Switching from Agalsidase Beta to Agalsidase Alfa in 10 Patients with Anderson-Fabry Disease

“…Numerous clinical trials, observational studies, and registry data have provided some evidence for the safety and efficacy of ERT in improving disease symptoms, cardiac mass, renal function, and quality of life Schiffmann et al 2001;Weidemann et al 2003;Wilcox et al 2004;Banikazemi et al 2007;Hughes et al 2008;Koskenvuo et al 2008;Imbriaco et al 2009;Mehta et al 2009;Feriozzi et al 2012). To date, there have been limited comparisons of the two agents, from which no firm conclusion can be drawn regarding their relative efficacy and safety (Lidove et al 2010).…”

Successful Management of Enzyme Replacement Therapy in Related Fabry Disease Patients with Severe Adverse Events by Switching from Agalsidase Beta (Fabrazyme®) to Agalsidase Alfa (Replagal®)

“…According to data from Shire HGT and Genzyme Corp held on file with the European Medicines Agency, 24% of men treated with agalsidase alfa develop IgG antibodies and 89% of patients treated with agalsidase beta develop IgG antibodies (European Medicines Agency 2006a, b). An IgE antibody response to ERT has only been reported for patients treated with agalsidase beta (Banikazemi et al 2007;Bodensteiner et al 2008), not in those receiving agalsidase alfa (Pastores et al 2007;Schiffmann et al 2006;Tesmoingt et al 2009). The frequency of infusion-related reactions is also higher with agalsidase beta than agalsidase alfa, at about 67% and 13.7%, respectively (European Medicines Agency 2006a, b).…”

Declaración de la Academia Chilena de Medicina sobre el Título V de la Ley 20.850 (Ley Ricarte Soto) y su proyecto de reglamento "De los ensayos clínicos de productos farmacéuticos y elementos de uso médico"

“…La literatura médica registra, precisamente, numerosos ensayos clínicos dirigidos a comprobar el beneficio de medicamentos de alto costo para la mayoría de las patologías seleccionadas en la ley 20.850 [4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20][21][22] .…”