Roxadustat is effective for correcting anemia in nondialysis CKD

  • Chen N & al.
  • N Engl J Med
  • 24 Jul 2019

  • International Clinical Digest
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Takeaway

  • Roxadustat bested placebo for correcting anemia in a phase 3 trial of patients with non-dialysis-dependent chronic kidney disease (NDD-CKD).

Why this matters

  • The hypoxia-inducible factor prolyl hydroxylase inhibitor represents a potential first-in-class treatment option.
  • Findings may herald a “revolution in the treatment of anemia in chronic kidney disease,” writes Joshua Kaplan, MD, in an accompanying editorial.

Study design

  • Double-blind, multicenter trial, 154 patients in China with NDD-CKD randomly allocated 2:1 to receive roxadustat or placebo 3x weekly for 8 weeks, followed by an 18-week open-label roxadustat extension period.
  • Mean baseline hemoglobin was 7-10 g/dL; 54% had transferrin saturation ≥20%; 38% had ferritin ≥200 μg/L.
  • Funding: FibroGen.   

Key results

  • Over weeks 7-9, mean hemoglobin increased 1.9±1.2 g/dL with roxadustat and decreased 0.4±0.8 g/dL with placebo (P<.001 style="list-style-type:circle;">
  • 67% achieved mean hemoglobin ≥10.0 g/dL (vs placebo, 6%).
  • At week 9, mean hepcidin reduction from baseline was greater with roxadustat vs placebo (56.14±63.40 vs 15.10±48.06 ng/mL), typically representing greater iron availability.
  • Reduction in total cholesterol from baseline was also greater with roxadustat vs placebo (−40.6 vs −7.7 mg/dL), representing a 23% decrease.
  • Adverse events with roxadustat included hyperkalemia and metabolic acidosis.
  • Roxadustat benefits were maintained through 18-week open-label period.
  • Limitations

    • Long-term safety unknown.

    Additional information