FDA Approves First Treatment for MCT8 Deficiency First therapy to bypass the broken transporter at the root of this very rare genetic disease and to decrease elevated blood thyroid hormone levels

https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-mct8-deficiency The U.S. Food and Drug Administration today approved Emcitate (tiratricol) tablets for oral suspension to treat peripheral thyrotoxicosis (excess thyroid hormone levels in the blood that causes symptoms, such as rapid heart rate, increased blood pressure and adverse effects on metabolism) in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. Emcitate is the first therapy approved by the FDA to treat symptoms of this very rare, devastating genetic disease. This approval reflects the FDA’s dedication to patients with rare genetic diseases, many of whom have serious unmet medical needs.