FDA Approves Tiratricol as First Treatment for Peripheral Thyrotoxicosis in MCT8 Deficiency

The FDA has approved tiratricol (Emcitate) for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency, also known as Allan-Herndon-Dudley syndrome. This is the first FDA-approved therapy for this rare genetic disorder, offering an option to address the chronic peripheral thyrotoxicosis that accompanies the disorder's severe neurodevelopmental impairment.