What Is Emcitate and Why Does It Matter?
Emcitate (tiratricol) is now approved by the FDA to treat a rare genetic condition called monocarboxylate transporter 8 (MCT8) deficiency. The condition, which mostly affects males, is also known as Allan-Herndon-Dudley syndrome.
In people with MCT8 deficiency, a rare genetic mutation prevents thyroid hormones from passing into their brain cells. Because of this, thyroid hormones build up in the blood. Too much thyroid hormone in the blood causes a condition called peripheral thyrotoxicosis. There are many symptoms caused by this condition, including a fast heart rate, high blood pressure, inability to tolerate heat, anxiety, fatigue, and severe weight loss.
Emcitate works like natural thyroid hormone. Unlike natural thyroid hormone, however, it can pass into brain cells in people with MCT8. This helps lower the high thyroid hormone levels in the blood.
Why Was Emcitate Approved?
The FDA approved Emcitate based on two clinical studies in adults and children with MCT8 deficiency. The most common side effects in both studies were diarrhea, vomiting, rash, and excessive sweating.
In one study of 20 male patients, 15 patients were split into two groups for 30 days after setting their dose. One group kept taking Emcitate, while the other group switched to a placebo, which is a dummy pill with no active medicine. Patients who switched to placebo saw their blood thyroid hormone levels jump up by an average of 64.6 ng/dL. Patients who stayed on Emcitate kept their levels steady.
In a second study, 46 male patients took Emcitate for one year. All patients had very high thyroid levels at the start of the study. Over 12 months of taking the medicine, thyroid levels dropped into the normal range. Patients also showed improvements in heart rate and blood pressure.
What Do I Need to Know About Taking Emcitate?
Emcitate comes as tablets that are taken one to three times daily. The tablets should not be swallowed or chewed. Instead, they should be mixed in drinking water to form a cloudy liquid suspension. This suspension can be swallowed by mouth or given through a feeding tube directly into the stomach. You should not mix the tablets with food, juice, or milk, but the suspension can be taken with or without food.
Do not stop taking Emcitate suddenly. Stopping quickly can make symptoms of high thyroid levels return or get worse. If you need to stop the medicine, your healthcare provider will lower the dose slowly. Tell your healthcare provider right away if you get new or worsening symptoms, such as a fast heart rate, high blood pressure, sweating, diarrhea, or trouble sleeping.
Tell your healthcare provider about all medicines, supplements, and vitamins you take. Do not take Emcitate with other thyroid drugs. Since iron and calcium supplements can lower how much Emcitate gets into your body, you should take these supplements consistently at the same time every day. Talk to your healthcare provider about the best way to schedule your doses.