Bayer HealthCare and Onyx Pharmaceuticals have announced that the US Food and Drug Administration (FDA) has granted priority review designation to the Supplemental New Drug Application (sNDA) for Nexavar® (sorafenib) tablets, for the treatment of locally advanced or metastatic radioactive iodine (RAI)-refractory differentiated thyroid cancer.
“We welcome this priority review as it supports our ongoing effort to make more treatment options available for cancer patients who until now have only limited or no treatment options,” said Kemal Malik, MD, Member of the Bayer HealthCare Executive Committee and Head of Global Development.
The FDA grants priority review to medicines that, if approved, would significantly improve the efficacy or safety of treatment for serious conditions. Under the Prescription Drug User Fee Act (PDUFA), the FDA aims to complete priority review within six months, rather than the standard ten-month review cycle.
The regulatory submission is based on data from the Phase III DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial, an international, multicentre, placebo-controlled trial. In the trial, sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study, 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 patients treated with sorafenib, compared to 5.8 months in patients receiving placebo.
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, diarrhoea, alopecia, rash/desquamation, fatigue, weight loss and hypertension. Results from the trial were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) in June 2013.
DECISION Trial Design
The DECISION trial was an international, multicentre, placebo-controlled study. A total of 417 patients with locally advanced or metastatic, RAI-refractory, differentiated thyroid cancer (papillary, follicular, Hürthle cell and poorly differentiated) who had received no prior chemotherapy, tyrosine kinase inhibitors, monoclonal antibodies that target VEGF or VEGF receptor, or other targeted agents for thyroid cancer were randomised to receive 400 mg of oral sorafenib twice daily (207 patients) or matching placebo (210 patients).