Egetis Therapeutics has announced the US launch of EMCITATE (tiratricol), a thyroid hormone receptor agonist indicated for the treatment of peripheral thyrotoxicosis in adults and paediatric patients with monocarboxylate transporter 8 (MCT8) deficiency, also known as Allan-Herndon-Dudley syndrome.
The commercial launch comes ahead of the anticipated timeline following the US Food and Drug Administration (FDA) approval of EMCITATE on September 28, 2026.
According to Egetis, EMCITATE is the first and only FDA-approved treatment for MCT8 deficiency in the United States.
“Launching EMCITATE in the United States ahead of our anticipated timelines marks an important milestone for people living with MCT8 deficiency, their families and everyone who has worked toward bringing an approved treatment to this community,” said Nicklas Westerholm, CEO of Egetis Therapeutics.
“Following FDA approval, our focus is now on building on this momentum and helping eligible patients access treatment as quickly as possible,” he added.
MCT8 deficiency is a rare, life-limiting, X-linked genetic disorder caused by pathogenic mutations in the SLC16A2 gene. These mutations impair the function of MCT8, a cell-surface transporter essential for thyroid hormone transport in specific cells, including in the brain.
The resulting disruption of thyroid hormone signalling leads to severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis. People with MCT8 deficiency have a reported median life expectancy of approximately 35 years.
To support the US launch, Egetis has established Egetis RareLink, a dedicated patient support programme developed in partnership with PANTHERx Rare Pharmacy. The programme is designed to help eligible patients, caregivers and healthcare professionals navigate medication access, treatment education, care coordination and ongoing support services.
Through Egetis RareLink, the company aims to facilitate access to EMCITATE and provide dedicated assistance throughout the treatment journey.
Egetis noted that EMCITATE is not recommended for the treatment of primary hypothyroidism.
