FDA Approves New Rx For Rare Cancer

Cometriq approved to treat advanced medullary thyroid cancer

/ Author:  / Reviewed by: Joseph V. Madia, MD

(RxWiki News) A new drug to treat advanced thyroid cancer that has spread to other areas of the body has been approved. The US Food and Drug Administration has approved Cometriq (cabozantinib) to treat medullary thyroid cancer.

Medullary thyroid cancer accounts for only about 4 percent of the 56,500 thyroid cancers each year, making it a fairly rare cancer.

It affects cells in the thyroid gland that makes a hormone called calcitonin, which helps keep healthy levels of calcium in the blood.

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“Cometriq is the second drug approved to treat medullary thyroid cancer in the past two years and reflects FDA’s commitment to the development and approval of drugs for treating rare diseases,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

The only other drug used to treat advanced medullary cancer is Caprelsa (vandetanib), which the FDA approved in April 2011.

FDA completed its 6-month priority review of Cametriq. The program speeds up the review process for drugs that either offer major treatment advancements or provide treatment options when no effective therapy exists.

This medication is what’s known as a kinase inhibitor. It blocks proteins that are involved in starting and growing medullary cancer cells.

A clinical trial involving 330 medullary cancer patients established the drug’s safety and effectiveness. Patients treated with Cometriq had longer periods of time before the disease got worse, which is known as progression-free survival. The drug also shrunk the size of tumors in 27 percent of patients. However, the drug did not extend the lives of patients.

Patients who received Cometriq lived about 11 months without disease progression, while the average was four months without progression for people who took a placebo (sugar pill). Reductions in tumor size lasted nearly 15 months compared to patients treated with placebo who saw no tumor shrinkage.

The prescribing information for Cometriq has a Boxed Warning alerting patients and healthcare professionals that fatal bleeding occurred in some patients, and that holes in the colon were also seen in some patients.

The most common side effects were diarrhea; inflammation or sores of the mouth; redness, pain or swelling of the digits (hand-foot syndrome); weight loss; nausea; loss of appetite; fatigue; oral pain; graying or loss of hair color; bad taste in the mouth; new or worsening high blood pressure; abdominal pain and constipation.

Other common abnormalities included increases in liver enzymes, low calcium and phosphorus and decreased white blood cells and platelets.

The only other drug used to treat advanced medullary cancer is Caprelsa (vandetanib), which the FDA approved in April 2011.

Cometriq is marketed by Exelixis, based in South San Francisco, Calif. Caprelsa is marketed by Wilmington, Del.-based AstraZeneca Pharmaceuticals.

Reviewed by: 
Review Date: 
November 29, 2012
Last Updated:
November 30, 2012