FDA approves vandetanib for rare thyroid cancer

FDA recently approved vandetanib to treat patients with late-stage (metastic) medullary thyroid cancer who are ineligible for surgery and have a disease that is growing and causing symptoms.

Symptoms of medullary thyroid cancer may include coughing, difficulty swallowing, enlargement of the thyroid gland, swelling of the neck, a lump on the thyroid, and hoarseness.

The approval of vandetanib, which targets medullary thyroid cancer’s ability to grow and expand, is an important move, since there are no other FDA-approved treatments for this type of cancer. Medullary thyroid cancer represents about 3% to 5% of all thyroid cancer cases, according to FDA.

At the same time, vandetanib was shown to cause irregular heartbeat in some patients, which could lead to death. As a result, only healthcare professionals and pharmacies certified through the vandetanib Risk Evaluation and Mitigation Strategy will be able to prescribe and dispense the drug.

FDA approved vandetanib based on a single randomized international study of 331 patients with late-stage medullary thyroid cancer. Patients who received vandetanib versus the placebo had a longer period of time without disease progression. Median progression-free survival was 16.4 months in the placebo arm and at least 22.6 months in the vandetanib arm.

However, during the study, 5 patients died from breathing complications, heart failure, and sepsis. Common side effects include diarrhea, rash, nausea, high blood pressure, headache, fatigue, decreased appetite, and abdominal pain.