Emcitate (tiratricol)

Tiratricol is the first FDA-approved treatment for MCT8 deficiency. The U.S. indication, approved on 28 September 2026, is peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency, and the label states that tiratricol is not recommended for primary hypothyroidism; the EU indication, authorised in February 2025, is peripheral thyrotoxicosis in MCT8 deficiency from birth. The efficacy evidence in the label consists of serum total T3 lowering in a 30-day randomized withdrawal study of 15 participants and a 12-month single-arm study of 46 participants, with secondary changes in heart rate, systolic blood pressure, and body weight. Triac Trial II did not meet its co-primary neurodevelopmental endpoints. Dosing is titrated to total T3 measured by LC-MS/MS, and the label states that no FDA-authorized LC-MS/MS test for total T3 is currently available. Egetis expects U.S. commercial availability eight to ten weeks after approval, with dispensing through PANTHERx Rare.