Oral Hypoxia-Inducible Factor Prolyl Hydroxylase Inhibitor Roxadustat (FG-4592) for Treatment of Anemia in Chronic Kidney Disease: A Placebo-Controlled Study of Pharmacokinetic and Pharmacodynamic Profiles in Hemodialysis Patients

J Clin Pharmacol. 2020 Nov;60(11):1432-1440. doi: 10.1002/jcph.1648. Epub 2020 Jun 30.

Abstract

Roxadustat (FG-4592), an oral hypoxia-inducible factor prolyl hydroxylase inhibitor that stimulates erythropoiesis, was evaluated in a phase 1b study in patients with end-stage renal disease with anemia on hemodialysis. Seventeen patients, on epoetin-alfa maintenance therapy with stable hemoglobin levels ≥10 g/dL, had epoetin-alfa discontinued on day 3 and were enrolled in this double-blind placebo-controlled study. Two cohorts were randomized 3:1 (roxadustat: placebo). Patients received single doses of roxadustat (1 or 2 mg/kg) or placebo 1 hour after hemodialysis on day 1 and 2 hours before dialysis on day 8. Maximum plasma concentration and area under the plasma concentration-time curve for patients receiving roxadustat were slightly more than dose proportional and elimination half-life ranged from 14.7 to 19.4 hours. Roxadustat was highly protein bound (99%) in plasma, and dialysis contributed a small fraction of the total clearance: only 4.56% and 3.04% of roxadustat recovered from the 1 and 2 mg/kg dose groups, respectively. Roxadustat induced transient elevations of endogenous erythropoietin that peaked between 7 and 14 hours after dosing and returned to baseline by 48 hours after dosing. Peak median endogenous erythropoietin levels were 96 mIU/mL and 268 mIU/mL for the 1- and 2-mg/kg doses, respectively, within physiologic range of endogenous erythropoietin responses to hypoxia at high altitude or after blood loss. No serious adverse events were reported, and there were no treatment- or dose-related trends in adverse event incidence.

Keywords: anemia; dialysis; erythropoietin; pharmacokinetics; roxadustat.

Publication types

  • Clinical Trial, Phase I
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Anemia / drug therapy*
  • Anemia / etiology
  • Area Under Curve
  • Dose-Response Relationship, Drug
  • Double-Blind Method
  • Erythropoiesis / drug effects
  • Erythropoietin / blood
  • Female
  • Glycine / administration & dosage
  • Glycine / adverse effects
  • Glycine / analogs & derivatives*
  • Glycine / blood
  • Glycine / pharmacokinetics
  • Humans
  • Hypoxia
  • Hypoxia-Inducible Factor-Proline Dioxygenases / antagonists & inhibitors*
  • Isoquinolines / administration & dosage*
  • Isoquinolines / adverse effects
  • Isoquinolines / blood
  • Isoquinolines / pharmacokinetics*
  • Kidney Failure, Chronic / complications*
  • Male
  • Middle Aged
  • Prolyl-Hydroxylase Inhibitors / administration & dosage*
  • Prolyl-Hydroxylase Inhibitors / adverse effects
  • Prolyl-Hydroxylase Inhibitors / blood
  • Prolyl-Hydroxylase Inhibitors / pharmacokinetics*
  • Renal Dialysis
  • Treatment Outcome

Substances

  • EPO protein, human
  • Isoquinolines
  • Prolyl-Hydroxylase Inhibitors
  • Erythropoietin
  • Hypoxia-Inducible Factor-Proline Dioxygenases
  • Glycine
  • roxadustat