ASN's Mission

To create a world without kidney diseases, the ASN Alliance for Kidney Health elevates care by educating and informing, driving breakthroughs and innovation, and advocating for policies that create transformative changes in kidney medicine throughout the world.

learn more

Contact ASN

1401 H St, NW, Ste 900, Washington, DC 20005


The Latest on X

Kidney Week

Please note that you are viewing an archived section from 2022 and some content may be unavailable. To unlock all content for 2022, please visit the archives.

Abstract: FR-PO231

Pharmacokinetic (PK) Results From a Phase 3 Trial to Evaluate Pegunigalsidase Alfa Every 4 Weeks (Q4W) in Patients (Pts) With Fabry Disease Previously Treated With Agalsidase Beta or Agalsidase Alfa

Session Information

  • Pharmacology
    November 04, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category: Pharmacology (PharmacoKinetics‚ -Dynamics‚ -Genomics)

  • 1900 Pharmacology (PharmacoKinetics‚ -Dynamics‚ -Genomics)


  • Tøndel, Camilla, Haukeland Universitetssjukehus, Bergen, Norway
  • Wallace, Eric L., University of Alabama, Birmingham, Alabama, United States
  • Rocco, Rossana, Chiesi Farmaceutici S.p.A., Parma, Italy
  • Almon, Einat, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Alon, Sari, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Blinder-Haddad, Liora, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Chertkoff, Raul, Protalix Biotherapeutics, Carmiel, Northern, Israel
  • Warnock, David G., University of Alabama, Birmingham, Alabama, United States
  • Waldek, Stephen, University of Sunderland, Sunderland, United Kingdom
  • Pisani, Antonio, University Federico II of Naples, Naples, Italy

Fabry disease is a lysosomal storage disease caused by X-linked deficiency of alfa-galactosidase-A. Two enzyme replacement therapies (ERTs) are approved for Fabry disease (agalsidase alfa and agalsidase beta) with infusion every 2 wks. We present PK results from a phase 3 trial of pegunigalsidase alfa administered Q4W to pts with Fabry disease previously treated with ERTs.


In this phase 3, open-label, switch-over study, pts aged 18–60y with Fabry disease who had received ERTs for ≥3y (with stable dose for ≥6mo) and had estimated glomerular filtration rate ≥30mL/min/1.73 m2 received IV infusion of pegunigalsidase alfa 2.0mg/kg Q4W for 52 wks. Blood samples for PK assessment were collected on Day 1 and at Wk 52 at 13 timepoints.


30 pts (24 male, 6 female) enrolled; 29 completed the study and entered the open-label extension (OLE) study. Main PK parameters are shown (Table). From baseline to week 52, pegunigalsidase alfa had a mean Cmax ranging from 35 876.7–46 829.6ng/mL, with tmax near end of infusion time, and mean t1/2 ranging from 100–134h. Detectable pegunigalsidase alfa concentrations after each 4-wk dosing interval were above the lower limit of quantitation (19.5ng/mL), ranging from 167.0–301.5ng/mL. Pts with antidrug antibodies (ADAs) at baseline (n=10) had lower AUC0-last (1 047 637h●ng/mL) and shorter t1/2 (41.0h; range: 1.1–136.3h) after 1st infusion and throughout the study vs the overall population.


Mean pegunigalsidase alfa concentration at the end of each dosing interval supported Q4W dosing. Long-term clinical outcomes with Q4W dosing are being assessed in the OLE.


  • Commercial Support –