EMA has recommended granting a marketing authorisation in the European Union (EU) for Emcitate (tiratricol), an oral treatment for peripheral thyrotoxicosis (inappropriately high levels of circulating thyroid hormones) in patients with monocarboxylate transporter 8 (MCT8) deficiency.
MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome (AHDS), is an ultra-rare, chronic and severely debilitating disease caused by mutations in the gene coding for the thyroid hormone transporter MCT8 protein. This prevents the thyroid hormones from entering cells in the brain and delays brain development in individuals with AHDS. This lack of thyroid hormone in the brain leads to severe intellectual and motor disability. Patients only rarely achieve independent sitting, and most will not be able to maintain head control…
