FDA Approves Drug Combination for Aggressive Type of Thyroid Cancer

The US Food and Drug Administration (FDA) has approved a combination of 2 targeted drugs to treat people with anaplastic thyroid cancer that has a certain type of gene mutation (BRAF V600E) and cannot be completely removed through surgery. The combination of Tafinlar (dabrafenib) and Mekinist (trametinib) is the first FDA-approved treatment for this type of thyroid cancer. This same drug combination was previously approved for BRAF mutation-positive melanoma skin cancer and non-small cell lung cancer that has spread to other parts of the body (metastasized).

Anaplastic thyroid cancer makes up only about 2% of all thyroid cancers. This new drug approval is significant, because this type of cancer can spread quickly and be very hard to treat. The FDA granted the approval using several programs designed to speed up the availability of drugs to treat serious diseases. They include priority review status, breakthrough therapy designation, and orphan drug designation.

The FDA based its approval on a study of 23 people with rare cancers that had the BRAF V600E mutation. More than half of patients (13) had a partial response, which is a way of saying their tumors shrank but did not go away completely. One patient had a complete response, which means no cancer remained detectable in the body. In 9 out of the 14 patients who had responses, the tumors had no significant growth for 6 months or longer.

Tafinlar and Mekinist are taken as pills or capsules. Common side effects can include skin changes, rash, itching, sensitivity to the sun, headache, fever, chills, joint or muscle pain, fatigue, cough, hair loss, nausea, diarrhea, and high blood pressure.

Less common but serious side effects can include bleeding, heart rhythm problems, liver or kidney problems, lung problems, severe allergic reactions, severe skin or eye problems, and increased blood sugar levels. Some people treated with these drugs develop skin cancers, especially squamous cell skin cancers.

Tafinlar and Mekinist are marketed by Novartis Pharmaceuticals Corporation.

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