​MCT8 deficiency had no approved US treatment until now

The FDA has approved Emcitate (tiratricol) as the first treatment for peripheral thyrotoxicosis in patients with the ultra-rare MCT8 deficiency, following evidence from six clinical studies that demonstrated its efficacy in normalizing serum T3 levels and alleviating symptoms. Tiratricol bypasses the defective MCT8 transporter by entering cells independently, addressing harmful peripheral thyrotoxicosis without affecting cerebral hypothyroidism.

This Wire brief sits within Fusion42's coverage of Biotech.