Updated Evaluation of Agalsidase Alfa Enzyme Replacement Therapy for Patients with Fabry Disease: Insights from Real-World Data

Abstract

The clinical use of agalsidase alfa as enzyme replacement therapy (ERT) for Fabry disease (FD) has spread since 2001, and a large body of evidence of its effectiveness has been collected. This review presents the clinical and laboratory results achieved with agalsidase alfa, which has been published in the literature. Agalsidase alfa infusion slows down or stops the progression of renal damage, expressed by reduction or stabilization of the annual decline of the glomerular filtration rate; yearly decrease of glomerular filtration rate (slope) sometimes is reduced until its stabilization. ERT prevents or reduces the occurrence of hypertrophic cardiomyopathy or slows the increase over time if it is already present. Moreover, regarding neurological manifestations, ERT improves neuropathic pain and quality of life, and recent data indicated that it may also prevent the burden of cerebrovascular disease. In addition to ERT’s clinical benefits, crucial topics like the most appropriate time to start therapy and the role of anti-drug antibodies (ADA) are analyzed. Treatment with agalsidase alfa in patients with FD substantially improves their outcomes and enhances their quality of life in patients with FD.

Keywords:

• Fabry disease
• agalsidase alfa
• enzyme replacement therapy
• clinical outcome in Fabry treated patients

Acknowledgments

The authors thank Mrs Vivienne Wall for the technical assistance in English.

Disclosure

Sandro Feriozzi has received travel assistance and honoraria for lecturing and participating in advisory boards from Sanofi, Takeda, Otsuka and Amicus. Cristina Chimenti has received travel assistance and honoraria for lecturing and participating in Pfizer, Sanofi, Alnylam, Takeda, and Amicus advisory boards. Ricardo Claudio Reisin has received travel assistance and honoraria for lecturing and participating in advisory boards from Sanofi, Takeda, PTC, Astra Zeneka, Pfizer and CSL Behring. The authors report no other conflicts of interest in this work.