The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended Emcitate (tiratricol) for treating peripheral thyrotoxicosis in patients with Allan-Herndon-Dudley syndrome, a rare genetic disorder caused by mutations in the MCT8 gene. Emcitate, designated as an orphan drug, can restore normal thyroid hormone activity in MCT8-dependent tissues. A phase 2 trial showed significant improvements in thyroid hormone levels and some secondary endpoints after 12 months of treatment. Adverse events were reported, including treatment-related side effects and serious complications like infections and death. Emcitate will be available in 350 µg dispersible tablets pending final approval by the European Commission.
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Emcitate Recommended for Peripheral Thyrotoxicosis in AHDS
