Trial Data Shows Promise in CABOMETYX for Differentiated Thyroid Cancer

In the past, Exelixis’ drug CABOMETYX (cabozantinib) has been approved for use in a variety of patients. For example, the treatment is approved for patients with renal cell carcinoma (RCC) as a monotherapy or in conjunction with nivolumab, as well as those with hepatocellular carcinoma. CABOMETYX may also be used for patients aged 12+ with locally advanced or metastatic differentiated thyroid cancer (DTC) whose condition has progressed on anti-VEGFR treatment.

According to a recent press release, there is now more positive information available on CABOMETYX for patients with differentiated thyroid cancer. In particular, the Phase 3 COSMIC-311 clinical trial highlighted the drug’s benefits for patients with previously treated DTC who are resistant to radioactive iodine treatment. 


During the clinical trial, researchers evaluated the impact of CABOMETYX on survival in patients with differentiated thyroid cancer. Altogether, 258 patients were treated. These patients received either 60mg CABOMETYX or a placebo once daily. The treatment is a tyrosine kinase inhibitor which is orally administered. Findings from the study, sourced from both the above press release and this separate news release, include:

  • In patients receiving the placebo, they survived without disease progression for a median of 1.9 months. Alternately, progression-free survival for patients taking CABOMETYX was 11 months, highlighting a huge improvement.
  • CABOMETYX showed promise for treating patients and had solid, promising outcomes, despite the patient’s prior treatment to other therapies.
  • At least one patient achieved a complete response during the trial’s follow-up period.
  • Ultimately, CABOMETYX showed promise in overall and objective response rates, progression-free and overall survival, and safety.
  • Although the treatment was found to be relatively safe and well-tolerated, some side effects did occur. These include fatigue, appetite loss, nausea and vomiting, and unintended weight loss. For a full list of CABOMETYX safety information, please take a look at the company’s website.

Thyroid Cancer

As the name suggests, thyroid cancer begins in the thyroid, a gland at the base of the throat. Normally, this gland produces hormones which control a variety of bodily processes and functions, such as blood pressure, body temperature, and even weight. However, when unregulated cellular growth occurs, tumors can form in the thyroid. Risk factors include a family history of thyroid cancer, radiation exposure (especially in the head and neck), and genetic diseases such as multiple endocrine neoplasia types 2a and 2B.

Multiple forms of thyroid cancer exist. These include papillary thyroid cancer, follicular thyroid cancer, medullary thyroid cancer, and anapestic thyroid cancer. In the above trial, researchers evaluated CABOMETYX for differentiated thyroid cancer, which is often slow-growing. Compared to other forms of cancer, differentiated thyroid cancer often affects slightly younger individuals, although pediatric cases are rare. Females are more likely to develop thyroid cancer than males. Unfortunately, differentiated thyroid cancer does not have a great prognosis, with many expected to survive only 3-5 years past diagnosis. Thus, new and innovative treatment options are urgently needed.

Symptoms include:

  • Painless lump in the neck
  • Difficulty breathing or swallowing
  • Neck and throat pain and inflammation
    • Note: This pain may extend up, even reaching the ears.
  • Chronic cough
  • Swollen lymph nodes
  • Voice changes, such as hoarseness

Learn more about thyroid cancer.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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