FDA Approves Sorafenib for Advanced Thyroid Cancer

November 26, 2013

The FDA has approved sorafenib (Nexavar) for the treatment of metastatic differentiated thyroid cancer.

The US Food and Drug Administration (FDA) has approved sorafenib (Nexavar), an oral kinase inhibitor, for metastatic, differentiated thyroid cancer (the most common type). It is the first agent to be approved for this form of thyroid cancer in 4 decades.

Sorafenib is indicated for patients with either locally recurrent or metastatic, progressive differentiated thyroid cancer that no longer responds to radioactive iodine treatment, the first-line therapy for thyroid cancer. The drug was approved based on a priority review by the FDA and received an orphan drug designation.

“Differentiated thyroid cancer can be challenging to treat, especially when unresponsive to conventional therapies,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “Today’s approval demonstrates the FDA’s commitment to expediting the availability of treatment options for patients with difficult-to-treat diseases.”

According to the National Cancer Institute, about 60,220 people in the United States will be diagnosed with thyroid cancer in 2013, and approximately 1,850 will die of the disease.

The approval of sorafenib is based on the 417-patient DECISION phase III clinical trial presented at the annual meeting of the American Society of Clinical Oncology (ASCO) in June 2013. Sorafenib increased progression-free survival by 41% compared with placebo. Patients in the sorafenib arm had a median progression-free survival of 10.8 months compared with 5.8 months on the placebo arm (P < .0001).

Ninety-six percent of patients on the trial had metastatic differentiated thyroid cancer that had progressed within the prior 14 months following treatment with radioactive iodine.

In the trial, the most common adverse events in sorafenib-treated patients were fatigue, infection, diarrhea, alopecia, rash, hand-foot skin reaction, nausea, weight loss, decreased appetite, abdominal and gastrointestinal pains, and hypertension.

Patients may need adjustment of their thyroid hormone replacement therapy as sorafenib is likely to increase levels of thyroid stimulating hormone in patients.

Sorafenib has been approved as a first-line treatment for advanced renal cell carcinoma since 2005 and as a first-line treatment of hepatocellular carcinoma since 2007.