FDA OKs Sorafenib for Advanced Thyroid Cancer

Nick Mulcahy


November 22, 2013

The US Food and Drug Administration (FDA) today approved sorafenib (Nexavar, Bayer) for the treatment of metastatic differentiated thyroid cancer.

The drug's new use is intended for patients with locally recurrent or metastatic, progressive differentiated thyroid cancer that no longer responds to radioactive iodine treatment.

Sorafenib becomes the first new drug for metastatic thyroid cancer in 40 years and opens up a new field in medical oncology, according to one of the drug's investigators, Marcia Brose, MD, PhD, an assistant professor of otolaryngology and head and neck surgery at the Abramson Cancer Center, University of Pennsylvania, in Philadelphia. She commented about the drug this year at the annual meeting of the American Society of Clinical Oncology® (ASCO®).

"Up until now, patients in this population haven't even come to medical oncology because there wasn't any treatment for them," she said.

"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 at the FDA's Center for Drug Evaluation and Research, in a press statement today.

At ASCO®, Dr. Brose explained that thyroid cancer has been treated generally by endocrinologists because medical oncology had no effective treatments once a patient became resistant to radioactive iodine.

The safety and effectiveness of sorafenib were established in a phase 3 clinical trial, known as DECISION, in 417 patients with progressive, radioactive, iodine-refractory differentiated thyroid cancer. The results showed a significant improvement in median progression-free survival to 10.8 months in the sorafenib group compared with 5.8 months in the placebo arm.

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, according to the FDA.

Sorafenib blocks the enzyme RAF kinase, which is a critical component of the RAF/MEK/ERK signaling pathway that controls cell division and proliferation, and it also inhibits the VEGFR-2/PDGFR-beta signaling cascade.

Sorafenib is also approved to treat hepatocellular carcinoma and advanced renal cell carcinoma.

The FDA approved sorafenib's new indication under its priority review program. This program provides for an expedited, 6-month review for drugs that may offer a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. Sorafenib also received orphan-product designation because it is intended to treat a rare disease or condition.

Dr. Brose has reported a financial relationship with Bayer.


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