First FDA-Approved Treatment
The FDA approved Emcitate (tiratricol), the first FDA-approved treatment for MCT8 deficiency, for peripheral thyrotoxicosis in patients with the rare genetic condition.
What The Approval Covers
Emcitate is approved as tablets for oral suspension to treat excess thyroid hormone in the bloodstream that causes symptoms.
About MCT8 Deficiency
MCT8 deficiency affects thyroid hormone transport into the brain. The FDA says the condition primarily affects males and is caused by a faulty gene.
How Emcitate Works
According to the FDA, tiratricol can enter cells without relying on MCT8, bypassing the transporter problem and lowering elevated blood thyroid hormone levels.
Findings From Clinical Studies
Two studies included patients from infants to adults. The FDA reported lower excess blood hormone levels and improvements in cardiovascular and metabolic symptoms.
Reported Symptoms And Effects
Reported improvements included systolic blood pressure and heart rate. MCT8 deficiency can also involve developmental and physical limitations, feeding difficulties, and heart and metabolic stress.
Side Effects And Medication Warning
Common side effects included diarrhea, vomiting, rash, and excessive sweating. Patients taking another thyroid medication should talk with a healthcare provider before starting Emcitate; the two should not be used together.
Read The FDA Announcement
Visit the FDA’s announcement for its full wording on Emcitate’s approved use, study findings, administration options, and medication warning.