After years without treatment: Drug approved for genetic disease affecting men

The U.S. Food and Drug Administration (FDA) has approved for the first time a drug treatment for MCT8 syndrome, a rare genetic disease that primarily affects men. The drug, Emcitate, reduces excess thyroid hormone in the blood and may reduce damage to the heart and metabolism, but does not cure the neurological damage.

The U.S. Food and Drug Administration (FDA) approved yesterday (Monday) the drug Emcitate for the treatment of MCT8 syndrome, a rare genetic disease that primarily affects men. This is the first approval of its kind in the United States for a drug treatment for the disease, after years in which no approved treatment existed. The drug, based on the active ingredient Tiratricol, works to reduce excess levels of thyroid hormone in the blood, a phenomenon characteristic of the disease that damages the heart and metabolism. The approval was based on two clinical studies, including an international multicenter randomized placebo-controlled trial, which showed improvement in heart function and blood pressure measures. However, the drug does not cure the genetic defect and does not correct the severe neurological damage associated with the disease. Common side effects included diarrhea, vomiting, rash, and excessive sweating. The drug was developed by Egetis Therapeutics. The U.S. approval does not indicate its availability in Israel.

After years without treatment: Drug approved for genetic disease affecting men