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Kidney Week

Abstract: PO0560

Pegunigalsidase Alfa, Novel Pegylated Enzyme Replacement Therapy, Evaluated in Fabry Patients with Progressing Kidney Disease: A Randomized Clinical Trial Study Design

Session Information

Category: CKD (Non-Dialysis)

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


  • Warnock, David G., UAB Medicine, Birmingham, Alabama, United States
  • Hughes, Derralynn, LSDU, Institute of Immunity and Transplantation, Royal Free London NHS Foundation Trust, London, United Kingdom
  • Alon, Sari, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Chertkoff, Raul, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Almon, Einat, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Schiffmann, Raphael, Baylor Institute of Metabolic Diseases, Baylor University Medical Center, Dallas, Texas, United States

Fabry disease (FD) is an X-linked multisystem lysosomal storage disorder, affecting males and females caused by the deficient of α-galactosidase-A (α-Gal-A) activity. Long-term disease manifestations include progressive renal failure, hypertrophic cardiomyopathy, cardiac rhythm disturbances, stroke, and death. Two enzyme replacement therapies (ERT) and oral chaperon therapy are commercially available. The clinical benefit of available treatments may not be as robust as anticipated, especially in the subset of males with ‘classic’ Fabry disease. In the context of ERT, a combination of factors including dose, dosing interval, presence of anti-drug antibodies, estimated glomerular filtration rate (eGFR), and age at the time of ERT initiation, and proteinuria could explain the less than optimal responses achieved by the currently available ERT. Pegunigalsidase alfa is a novel PEGylated homo-dimer ERT which is more stable, has a favorable safety profile, potentially less development of anti-drug antibodies, and enhanced pharmacokinetic profile (~80 hours half-life and higher AUC) compared to other available ERT.


Adult FD patients (males and females) deteriorating in kidney function with annualized eGFR ≤ -2 mL/min/1.73 m2/year while on agalsidase beta have been enrolled into BALANCE, a phase-III double-blind active control study (NCT02795676), and were randomized (2:1 ratio) to pegunigalsidase alfa or continue agalsidase beta for 2 years at 1 mg/kg every other week. The primary outcome is the difference in the mean annualized slope of eGFR during the study between the two groups.


Description of the baseline characteristics for approximately 75 patients enrolled at 29 US and European study sites by: age, sex, enzymatic activity, genetic mutations, FD symptoms, previous FD treatment length, kidney function (eGFR, eGFR slope and UPCR), Lyso-Gb3, and anti-drug antibodies pre-treatment status.


The current work describes the design and methods of the study protocol and the baseline characteristics for approximately 75 enrolled patients in the study.


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