Radium Ra 223 dichloride injection: U.S. Food and Drug Administration drug approval summary

Clin Cancer Res. 2014 Jan 1;20(1):9-14. doi: 10.1158/1078-0432.CCR-13-2665. Epub 2013 Nov 4.

Abstract

On May 15, 2013, the U.S. Food and Drug Administration (FDA) approved radium Ra 223 dichloride (Ra-223; Xofigo injection; Bayer HealthCare Pharmaceuticals Inc.) for the treatment of patients with castration-resistant prostate cancer (CRPC), symptomatic bone metastases, and no known visceral metastatic disease. The FDA review was based on clinical trial BC1-06, which randomly allocated patients (2:1) to either Ra-223 plus best standard of care (BSoC) or placebo plus BSoC. The primary endpoint was overall survival (OS) with a key secondary endpoint of time to first symptomatic skeletal event (SSE). A statistically significant improvement in OS was demonstrated [HR, 0.70; 95% confidence interval, 0.55-0.88, P = 0.0019]. At the prespecified interim analysis, the median OS durations were 14.0 and 11.2 months in the Ra-223 and placebo arms, respectively. The improvement in OS was supported by a delay in time to first SSE favoring the Ra-223 arm. The most common (>10%) adverse reactions in patients receiving Ra-223 were nausea, diarrhea, vomiting, and peripheral edema. The most common (>10%) hematologic laboratory abnormalities were anemia, lymphocytopenia, leukopenia, thrombocytopenia, and neutropenia. Ra-223 is the first α-emitting radiotherapeutic and the first radiopharmaceutical to demonstrate an OS advantage in metastatic prostate cancer.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial

MeSH terms

  • Aged
  • Animals
  • Bone Neoplasms / mortality
  • Bone Neoplasms / radiotherapy*
  • Bone Neoplasms / secondary
  • Double-Blind Method
  • Drug Approval
  • Humans
  • Kaplan-Meier Estimate
  • Male
  • Prostatic Neoplasms, Castration-Resistant / mortality
  • Prostatic Neoplasms, Castration-Resistant / pathology
  • Prostatic Neoplasms, Castration-Resistant / radiotherapy*
  • Radioisotopes / adverse effects
  • Radioisotopes / therapeutic use
  • Radiopharmaceuticals / adverse effects
  • Radiopharmaceuticals / therapeutic use*
  • Radium / adverse effects
  • Radium / therapeutic use*
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration

Substances

  • Radioisotopes
  • Radiopharmaceuticals
  • radium Ra 223 dichloride
  • Radium