Bayer HealthCare Pharmaceuticals and Onyx Pharmaceuticals, Inc. have announced positive data from the Phase III DECISION trial investigating the use of sorafenib (Nexavar®) tablets in patients with locally advanced or metastatic radioactive iodine (RAI)-refractory differentiated thyroid cancer.
Sorafenib significantly extended progression-free survival (PFS) compared to placebo (HR=0.587 [95% CI, 0.454–0.758]; p<0.0001), which represents a 41% reduction in the risk of progression or death for patients who received sorafenib compared to placebo-treated patients. The median PFS was 10.8 months in sorafenib-treated patients compared to 5.8 months in those taking placebo. PFS as defined by Response Evaluation Criteria in Solid Tumors (RECIST 1.0) was the primary endpoint of this study. These data will be presented in a plenary session on June 2, 2013, at the 49th Annual Meeting of the American Society of Clinical Oncology (ASCO) in Chicago, IL (USA) (Abstract #4).
“While most patients with differentiated thyroid cancer are cured, there is a major treatment gap for patients whose cancer no longer responds to standard therapies, suffer from progression and ultimately die of their disease,” said Marcia Brose MD PhD, principal investigator of the DECISION trial and Assistant Professor in the Abramson Cancer Center and the Perelman School of Medicine at the University of Pennsylvania. “The DECISION trial results demonstrate for the first time that sorafenib may extend the progression free period for thyroid cancer patients.”
“Already the standard of care in HCC and an important treatment option in RCC, sorafenib’s utility may also be extended to differentiated thyroid cancer given these positive trial results,” said Kemal Malik MD, Member of the Bayer HealthCare Executive Committee and Head of Global Development. “This trial is representative of our continued commitment to fully understanding sorafenib’s applicability across tumour types, especially in hard-to-treat cancers where there are limited treatment options.”
There was no statistically significant difference in overall survival between treatment arms, which was expected due to the cross-over design of the study. After progression, patients receiving placebo had the option to begin open-label sorafenib at the discretion of the investigator; 71% of placebo patients ultimately began open-label sorafenib during the trial. Median overall survival has not yet been reached in either arm.
Safety and tolerability in the study were generally consistent with the known profile of sorafenib. The most common treatment-emergent adverse events in the sorafenib arm were hand-foot skin reaction, diarrhoea, alopecia, rash/desquamation, fatigue, weight loss and hypertension.
Bayer plans to submit the Phase III DECISION data as the basis to apply for marketing authorisation of sorafenib in the treatment of RAI-refractory differentiated thyroid cancer.