Emcitate is approved to treat peripheral thyrotoxicosis in adults and children with MCT8 deficiency, also known as Allan–Herndon–Dudley syndrome. The disease is serious, life-limiting, and X-linked, so it mainly affects boys. It is caused by mutations in the SLC16A2 gene, which disrupt the transport of thyroid hormone into certain cells, including brain cells. As a result, patients have too little thyroid hormone activity in the central nervous system and too much of the active hormone T3 in peripheral tissues. This leads to severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis. The reported median life expectancy is around 35 years.

A turning point

The approval was based on a clinical development program that included the ReTRIACt, Triac Trial I, and Triac Trial II studies, two Erasmus Medical Center studies, and a US expanded access program. Emcitate is already approved in the EU.

“Today marks a turning point for patients living with MCT8 deficiency and their caregivers, who have waited long for an approved treatment in the US. Our immediate focus is ensuring that eligible patients can access Emcitate as quickly as possible,” says Nicklas Westerholm, CEO, Egetis Therapeutics.

Commercially available in the US in eight to ten weeks after approval

Egetis expects Emcitate to be commercially available in the US in eight to ten weeks after approval. The company is launching a patient support program, Egetis RareLink, together with the specialty pharmacy PANTHERx Rare. Egetis plans to explore selling the priority review voucher, which could happen in the fourth quarter of 2026, depending on market conditions.