WASHINGTON, D.C. — The U.S. Food and Drug Administration approved Emcitate, or tiratricol, as the first therapy for peripheral thyrotoxicosis in patients with MCT8 deficiency, establishing a treatment option for a rare genetic disorder that can cause severe neurological, cardiovascular and metabolic complications.
The approval was granted to Egetis Therapeutics US Inc. and covers Emcitate tablets prepared as an oral suspension.
MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome, primarily affects males and is caused by a genetic defect involving the MCT8 transporter, a protein responsible for carrying thyroid hormone into the brain.
When the transporter does not function properly, insufficient thyroid hormone reaches the brain while excessive amounts accumulate in the bloodstream. Patients can experience intellectual disability, severely limited or absent speech, feeding problems and an inability to sit or walk independently, along with cardiovascular and metabolic complications.
Emcitate is designed to address the excess circulating thyroid hormone without relying on the defective transporter. Its active ingredient, tiratricol, can enter cells independently of MCT8.
“This drug sidesteps that problem, as its active ingredient, tiratricol, can enter cells on its own without relying on the broken transporter,” Hylton V. Joffe, director of the FDA’s Office of Cardiology, Hematology, Endocrinology, and Nephrology, said.
The FDA evaluated Emcitate in two clinical studies involving patients ranging from infants to adults. The research included an international, multicenter, randomized, placebo-controlled trial and a longer-term open-label study.
Across the studies, treatment reduced excessive thyroid hormone levels in the bloodstream and improved cardiovascular and metabolic measures affected by thyroid levels, including systolic blood pressure and heart rate.
Emcitate is administered once daily as a liquid suspension, either orally or through a feeding tube for patients who have difficulty swallowing.
The most common side effects were diarrhea, vomiting, rash and excessive sweating. The FDA cautioned that patients taking another thyroid medication should consult their health care provider before beginning Emcitate because the therapies should not be used together.
The agency previously granted the drug Orphan Drug, Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations, as well as Priority Review.
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