Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Summary

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Timeline

Start

2018-11-13

Primary completion

2026-04-13

Completion

2026-04-13

Publications

• Holida M, Linhart A, Longo N, Wallace E, Tondel C, Hughes D, Warnock DG, Pisani A, Eyskens F, Deegan P, Feldt-Rasmussen U, Goker-Alpan O, Mehta A, Piotti G, Fichera V, Wang M, Chertkoff R, Waldek S, Wilcox WR, Bernat JA. Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study. Orphanet J Rare Dis. 2026 Mar 20;21(1):185. doi: 10.1186/s13023-026-04303-8.

Drugs

Indications