Egetis Therapeutics has received FDA approval for the first drug to treat peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency, also called Allan–Herndon–Dudley syndrome (AHDS).
MCT8 deficiency is a rare, life-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2 gene. The mutations impair the function of MCT8, a critical cell-surface thyroid-hormone transporter responsible for thyroid hormone transport in specific cells, including in the brain.
Emcitate (tiratricol) is a thyroid hormone receptor agonist that treats disrupted thyroid hormone signaling by increasing activity in the central nervous system. The oral drug will be available to adults and pediatric patients. Along with approval for the drug, the FDA has granted Egetis a Rare Pediatric Disease Priority Review Voucher (PRV), which will enable the company to accelerate the review of a future application.
To help patients access Emcitate, Egetis has partnered with PANTHERx Rare to launch a comprehensive patient support program called Egetis RareLink. The program will support education, care coordination, and treatment for patients.