A phase III trial of sorafenib (Nexavar) in patients with locally advanced or metastatic radioactive iodine (RAI)-refractory differentiated thyroid cancer met its primary endpoint of a statistically significant improvement in progression-free survival (PFS) compared with a placebo.
Though no data have yet been made available, a joint announcement from Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals, Inc. reported that the results were positive, and detailed efficacy and safety analyses from this study are expected to be presented at a future medical meeting.
The phase III DECISION trial enrolled 417 patients in an international, multicenter, randomized, placebo-controlled study. Patients in the study had locally advanced or metastatic disease and had received no prior chemotherapy, tyrosine kinase inhibitors, monoclonal antibodies that target VEGF or VEGF receptor, or other targeted therapies for thyroid cancer.
The trial was initiated based on a series of positive phase II trials. For example, in the results of a single-arm trial presented at the 2011 annual meeting of the American Society of Clinical Oncology, the median PFS was 93.6 weeks and the median overall survival (OS) was 140.6 weeks in patients with progressing advanced, iodine-refractory differentiated or poorly differentiated thyroid cancer and medullary and anaplastic thyroid carcinoma who received 400 mg of sorafenib twice daily.
“Effective treatment options are urgently needed for patients with RAI-refractory differentiated thyroid cancer,” said Dimitris Voliotis, MD, vice president of Global Clinical Development Oncology for Bayer HealthCare, in a statement. “We are pleased that the results of this study demonstrate that Nexavar may provide a treatment option for these patients.”
Only a small number of drugs have been approved to treat thyroid cancer. Most recently, cabozantinib (Cometriq) was approved to treat patients with medullary thyroid cancer, and vandetanib (Caprelsa) was also approved for this indication in 2011. In the statement, Bayer noted that the results of this study would be used as the basis for marketing authorization of sorafenib in the treatment of patients with RAI-refractory differentiated thyroid cancer.