Bayer HealthCare and Onyx Pharmaceuticals recently announced that the FDA has granted priority review designation to the supplemental new drug application of sorafenib (Nexavar) tablets for the treatment of locally advanced or metastatic radioactive iodine-refractory differentiated thyroid cancer.
The regulatory submission is based on data from the phase III DECISION trial,1 an international, multicenter, placebo-controlled investigation of sorafenib in patients with locally advanced or metastatic radioactive iodine–refractory thyroid cancer.
The DECISION trial included a total of 417 patients with locally advanced or metastatic, radioactive iodine–refractory, differentiated thyroid cancer (papillary, follicular, Hürthle cell, and poorly differentiated) who had received no prior chemotherapy, tyrosine kinase inhibitors, monoclonal antibodies that target vascular endothelial growth factor (VEGF) or VEGF receptor, or other targeted agents for thyroid cancer. Patients were randomly assigned to receive 400 mg of oral sorafenib twice daily (207 patients) or matching placebo (210 patients).
In the DECISION trial, sorafenib significantly extended progression-free survival, the primary endpoint of the study, compared to placebo (hazard ratio = 0.587, 95% confidence interval = 0.454–0.758, P < .0001), which represents a 41% reduction in the risk of disease progression or death for patients who received sorafenib compared to placebo-treated patients.
The median progression-free survival was 10.8 months in patients treated with sorafenib, compared to 5.8 months in patients receiving placebo.
Safety and Tolerability
The safety and tolerability profile of sorafenib for patients in the trial was generally consistent with the known profile of sorafenib. The most common treatment-emergent adverse events in the sorafenib arm were hand-foot skin reaction, diarrhea, alopecia, rash/desquamation, fatigue, weight loss, and hypertension.
Results from the trial were presented at ASCO’s Annual Meeting in June 2013. ■
1. Brose MS, Nutting C, Jarzab B, et al: Sorafenib in locally advanced or metastatic patients with radioactive iondine-refractory differentiated thyroid cancer: The phase III DECISION trial. 2013 ASCO Annual Meeting. Abstract 4. Presented June 4, 2013.