The U.S. Food and Drug Administration recently expanded the approved uses of sorafenib (Nexavar) to treat late-stage differentiated thyroid cancer. The new indication is for patients with locally recurrent or metastatic, progressive differentiated thyroid cancer that no longer responds to radioactive iodine treatment.
“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. “[The recent] approval demonstrates the FDA’s commitment to expediting the availability of treatment options for patients with difficult-to-treat diseases.”
Clinical Trial Results
The safety and effectiveness of sorafenib were established in a clinical study involving 417 participants with locally recurrent or metastatic, progressive differentiated thyroid cancer that does not respond to radioactive iodine treatment. Treatment with sorafenib resulted 41% reduction in the risk of disease progression or death. The median progression-free survival was 10.8 months in patients treated with sorafenib, compared to 5.8 months in patients receiving placebo.
The most common side effects in patients treated with sorafenib were diarrhea, fatigue, infection, alopecia, hand-foot skin reaction, rash, weight loss, decreased appetite, nausea, gastrointestinal and abdominal pains, and hypertension. Thyroid-stimulating hormone, a potential promoter of thyroid cancer, is more likely to become elevated while on treatment with sorafenib, requiring adjustment of thyroid hormone replacement therapy.
The FDA completed its review of sorafenib’s new indication under its priority review program. Sorafenib also received orphan-product designation by the FDA because it is intended to treat a rare disease or condition.
The FDA approved sorafenib to treat advanced kidney cancer in 2005. In 2007, the agency expanded the drug’s label to treat liver cancer that cannot be surgically removed. ■
