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Abstract: PO0562

Switching from Agalsidase Alfa to Pegunigalsidase Alfa for Treating Fabry Disease: One Year of Treatment Data from Bridge, a Phase 3 Open-Label Study

Session Information

Category: CKD (Non-Dialysis)

  • 2102 CKD (Non-Dialysis): Clinical, Outcomes, and Trials

Authors

  • Tøndel, Camilla, Haukeland University Hospital, Bergen, Norway
  • Linhart, Ales, Charles University, Praha, Czechia
  • Dostálová, Gabriela, Charles University, Praha, Czechia
  • Nicholls, Kathleen M., The Royal Melbourne Hospital, Melbourne, Victoria, Australia
  • West, Michael L., Dalhousie University, Halifax, Nova Scotia, Canada
  • Jovanovic, Ana, Salford Royal, Dept of Inherited Metabolic Disease, Salford, United Kingdom
  • Giraldo, Pilar, Hospital de Dia Quiron, Zaragoza, Spain
  • Vujkovac, Bojan, General Hospital Slovenj Gradec, Slovenj Gradec, Slovenia
  • Almon, Einat, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Alon, Sari, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Szlaifer, Mali, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Chertkoff, Raul, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Hughes, Derralynn, LSDU, Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust, London, United Kingdom
Background

Pegunigalsidase-alfa is a novel, PEGylated α-Galactosidase-A enzyme in development for the treatment of Fabry disease (FD).

Methods

Bridge (PB-102-F30, NCT03018730) is a phase III, open-label, switch-over study, designed to assess the safety and efficacy of pegunigalsidase-alfa (1 mg/Kg EOW) in adult FD patients previously treated with agalsidase alfa for at least 2 years.

Results

This is an interim report of 12-months on-treatment data generated from the first 16 patients (9 males and 7 females) out of the 22 adult patients enrolled. Baseline characteristics: age 24-60 years, the mean estimated Glomerular Filtration Rate (eGFR) 75.45 in males and 85.78 mL/min/1.73m2 in females, annualized eGFR slope was -5.04 and -5.18 mL/min/1.73m2/year, respectively, mean residual leucocytes enzymatic activity 5.9% of lab normal mean in males and 27.9% in females, and plasma lyso-Gb3 53.6 and 13.8 nM, respectively. After one year the mean annualized eGFR slope improved from -5.10 mL/min/1.73m2/year while on agalsidase alfa, to -0.23 mL/min/1.73m2/year on pegunigalsidase-alfa. According to Wanner et al. 2018, FD patients with eGFR slope between ≥ -5 and < -3 mL/min/1.73 m2/year are defined as kidney disease progressing and with eGFR slope < -5 mL/min/1.73 m2/year are defined fast progressing. The therapeutic goal is to reach eGFR slope ≥ -3 mL/min/1.73 m2/year for the progressing, and ≥ -5 mL/min/1.73m2/year or more than 50% decrease in progression for the fast progressing. In this interim analysis, 100% of the progressing patients and 66.7% in the fast progressing group achieved the proposed therapeutic goals after switching to pegunigalsidase-alfa. The switch to pegunigalsidase-alfa was safe and well-tolerated. The majority of the patients who completed the study rolled over to a long-term extension study, continuing receiving pegunigalsidase-alfa.

Conclusion

These results suggest a potential benefit of pegunigalsidase-alfa on renal function for FD patients currently treated with agalsidase alfa, to be confirmed by long-term data.

Funding

  • Commercial Support