To announce the FDA approval of tiratricol for treating peripheral thyrotoxicosis in patients with MCT8 deficiency.
Approach:
FDA Approval: The FDA approved tiratricol (Emcitate) tablets for oral suspension for treating peripheral thyrotoxicosis in MCT8 deficiency.
Mechanism of Action: Tiratricol can enter cells independently of the MCT8 transporter, reducing elevated thyroid hormone levels in the blood.
Clinical Studies: Two clinical studies, including a randomized placebo-controlled trial and a long-term open-label study, demonstrated the efficacy of tiratricol.
