The US Food and Drug Administration (FDA) has approved Emcitate (tiratricol) for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency.
MCT8 deficiency is a rare disorder of brain development that causes moderate to severe intellectual disability and problems with movement. This condition, which occurs exclusively in males, is caused by mutations in the SLC16A2 gene. These mutations impair MCT8 function, a protein responsible for thyroid hormone transport in specific cells, including in the brain. As a result, patients experience disrupted thyroid hormone signaling, characterized by insufficient thyroid hormone activity in the central nervous system and excessive exposure to the active thyroid hormone triiodothyronine (T3) in peripheral tissues, with the latter leading to persistent peripheral thyrotoxicosis. Common symptoms include hypotonia, muscle hypoplasia, joint contractures, abnormal muscle stiffness, muscle weakness, and involuntary movements of the arms and legs.
Tiratricol is a thyroid hormone receptor agonist. It is the first FDA-approved treatment option for patients with MCT8 deficiency in the United States.
The approval is based on data from the clinical development program evaluating tiratricol in patients with MCT8 deficiency, including ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center (EMC) Cohort Study, EMC Survival Study and the US Expanded Access Program.
The effectiveness of tiratricol was evaluated in two clinical trials in patients ranging in age from infants to adults, including the international, multi-center, randomized, placebo-controlled ReTRIACt study and a longer-term open-label study. Across both studies, patients treated with tiratricol experienced reductions in excess thyroid hormone levels in the bloodstream and improvements in cardiovascular and metabolic symptoms impacted by thyroid levels, such as systolic blood pressure and heart rate. The most common side effects were diarrhea, vomiting, rash and excessive sweating.
For more information, visit https://www.egetis.com/news/egetis-therapeutics-announces-u-s-fda-approval-of-emcitate-tiratricol-for-patients-with-mct8-deficiency/
To learn more about MCT8 deficiency and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/
