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Abstract: TH-OR04

Roxadustat Is Not Associated with an Increased Risk of Neoplasm in Patients with CKD and Anemia

Session Information

Category: Anemia and Iron Metabolism

  • No subcategory defined

Authors

  • Coyne, Daniel W., Washington University in Saint Louis School of Medicine, Saint Louis, Missouri, United States
  • Fishbane, Steven, Northwell Health, Great Neck, New York, United States
  • Pergola, Pablo E., Renal Associates PA, San Antonio, Texas, United States
  • Leong, Robert, FibroGen Inc, San Francisco, California, United States
  • Zhong, Ming, FibroGen Inc, San Francisco, California, United States
  • Little, Dustin J., AstraZeneca, Gaithersburg, Maryland, United States
  • Yu, Kin-Hung Peony, FibroGen Inc, San Francisco, California, United States
Background

Roxadustat is a novel, orally bioavailable, small molecule that reversibly inhibits hypoxia-inducible factor (HIF) prolyl hydroxylase enzymes and activates HIF and transcription of HIF-responsive genes, including for erythropoietin. Preclinical studies of roxadustat in multiple animal species did not demonstrate a carcinogenic signal. We report neoplasm-related adverse events (AEs) and serious adverse events (SAEs) from the roxadustat global phase 3 program.

Methods

Data from six pivotal studies were pooled: 3 compared roxadustat to placebo in patients with non–dialysis-dependent (NDD) CKD, and 3 compared roxadustat to epoetin alfa in patients with dialysis-dependent (DD) CKD. Patients were excluded from the studies if they had a history of malignancy, except for those determined to be cured or in remission for ≥5 years, curatively resected basal/squamous cell cancers, cervical cancer in situ, or resected colon polyps. AEs/SAEs reported during the treatment period +28 days and +7 days after the last dose of study drug in the NDD- and DD-CKD populations, respectively, were compared. Events were coded using the Medical Dictionary for Regulatory Activities, System Organ Class “Neoplasms benign, malignant and unspecified (incl cysts and polyps).”

Results

In the NDD population, 4270 patients were randomized (roxadustat=2386, placebo=1884), corresponding to 3870.7 and 2323.2 patient-exposure years (PEY), respectively. Neoplasm-related AE rates were 2.5/100 PEY in both the roxadustat and placebo groups. Neoplasm-related SAE rates were 1.1/100 PEY and 1.3/100 PEY in the roxadustat and placebo groups. In the DD population, 3880 patients were randomized (roxadustat=1940, epoetin alfa=1940), corresponding to 3315.3 and 3743.6 PEY, respectively. Neoplasm-related AE rates were 2.7/100 PEY and 2.3/100 PEY in the roxadustat and epoetin alfa groups. Neoplasm-related SAE rates were 1.1/100 PEY and 1.2/100 PEY. In both the NDD- and DD-CKD populations, there were no between–treatment-group differences in the organ types of neoplasms.

Conclusion

There were no clinically meaningful between–treatment-group differences in neoplasm-related AE and SAE rates in the roxadustat phase 3 clinical trials.

Funding

  • Commercial Support