FDA approves emcitate for MCT8 deficiency

On September 28, 2026, the FDA approved Emcitate (tiratricol) to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The agency calls it the first approved therapy for symptoms of this very rare genetic disease. The indication covers the peripheral thyrotoxic symptoms of MCT8 deficiency.