China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Summary

This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

Timeline

Start

2021-10-13

Primary completion

2023-03-09

Completion

2023-03-09

Publications

• Ren H, Zhang W, Ouyang Y, Guo J, Xu H, Ma J, Luo X, Pan X, Yuan Y, Zhang W, Shen Q, Li B, Feng Q, Liu S, Chen N. A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease. Orphanet J Rare Dis. 2025 Aug 4;20(1):401. doi: 10.1186/s13023-025-03950-7.

Drugs

Indications