Bayer HealthCare and Onyx Pharmaceuticals, Inc, an Amgen subsidiary (Nasdaq: AMGN), has announced that the US Food and Drug Administration (FDA) has approved the oral multi-kinase inhibitor Nexavar® (sorafenib) for the treatment of patients with locally recurrent or metastatic, progressive, differentiated thyroid carcinoma (DTC) that is refractory to radioactive iodine (RAI) treatment. Nexavar was approved following a priority review by the FDA, a designation reserved for drugs that offer a significant improvement in treatment over existing options.
“Nexavar is an important treatment option for patients with certain kinds of cancers, and this latest indication for RAI-refractory differentiated thyroid cancer addresses a serious unmet medical need,” said Kemal Malik, MD, Member of the Bayer HealthCare Executive Committee and Head of Global Development. “Bayer is committed to exploring the full clinical potential of our therapies to make a difference in the lives of patients and physicians.”
“In the DECISION trial, patients with this type of advanced thyroid cancer who were treated with sorafenib nearly doubled their time to disease progression or death compared to patients treated with placebo,” said Martin Schlumberger, MD, of Institut Gustave-Roussy in Villejuif, France and co-lead investigator of the DECISION trial. “We are pleased that patients in the United States now have a new and noteworthy treatment option.”
DECISION trial design
The FDA approval 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 study. A total of 417 patients with locally advanced or metastatic, progressive, 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). Ninety-six percent of randomised patients had metastatic disease.
In the trial, sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study, compared to placebo (HR=0.59 [95% CI, 0.46-0.76]; p<0.001), 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 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 in 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, weight loss, fatigue, hypertension and rash. Results from the trial were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) in June 2013.
About thyroid cancer
Thyroid cancer is the most common endocrine malignancy. There are more than 213,000 new cases of thyroid cancer annually and approximately 35,000 people die from thyroid cancer worldwide each year.
Papillary, follicular (including Hürthle cell) and poorly differentiated types of thyroid cancer are classified as ‘differentiated thyroid cancer’ and account for approximately 94% of all thyroid cancers. While the majority of differentiated thyroid cancers are treatable, RAI-refractory locally advanced or metastatic disease is more difficult to treat and is associated with a lower patient survival rate.
