The FDA has approved Emcitate (tiratricol) to treat peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome.
Emcitate is the first FDA-approved treatment for peripheral thyrotoxicosis in patients with the rare genetic disorder, according to a Sept. 28 news release. The drug’s active ingredient, tiratricol, can enter cells without relying on the MCT8 transporter.
The drug’s effectiveness was evaluated in two clinical studies involving patients ranging from infants to adults. Across both studies, patients treated with Emcitate had reductions in excess thyroid hormone levels and improvements in cardiovascular and metabolic effects associated with elevated thyroid hormone levels, including systolic blood pressure and heart rate.
Emcitate is taken once daily as a liquid suspension by mouth or through a feeding tube. The FDA granted the approval to Egetis Therapeutics US.
