Roxadustat for Treating Anemia in Patients with CKD Not on Dialysis: Results from a Randomized Phase 3 Study

Area of Science:

• Nephrology
• Hematology
• Pharmacology

Background:

• Anemia in non-dialysis-dependent chronic kidney disease (CKD) often requires injectable therapies.
• Roxadustat offers an oral alternative, targeting hypoxia-inducible factor prolyl hydroxylase to stimulate erythropoiesis and improve iron metabolism.

Purpose of the Study:

• To evaluate the efficacy and safety of oral roxadustat compared to placebo in patients with non-dialysis-dependent CKD and anemia.
• To assess roxadustat's effect on hemoglobin levels and the need for red blood cell transfusions.

Main Methods:

• A double-blind, randomized phase 3 study involving 2781 patients with non-dialysis-dependent CKD stages 3-5 and hemoglobin <10.0 g/dl.
• Patients received thrice-weekly 70-mg oral roxadustat or placebo, with dose titration based on hemoglobin levels.
• Primary endpoint: mean change in hemoglobin from baseline averaged over weeks 28-52 versus placebo.

Main Results:

• Roxadustat significantly increased mean hemoglobin by 1.75 g/dl versus 0.40 g/dl for placebo (P<0.001).
• The treatment reduced the risk of red blood cell transfusion by 63% (HR, 0.37).
• Common adverse events included end-stage kidney disease (ESKD), urinary tract infection, pneumonia, and hypertension, with similar rates between groups.

Conclusions:

• Roxadustat is effective in increasing hemoglobin levels for anemic patients with non-dialysis-dependent CKD.
• The oral agent reduces the requirement for red blood cell transfusions.
• Roxadustat demonstrated an adverse event profile comparable to placebo.