Stockholm, Sweden, February 13, 2025. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), today announced that the European Commission (EC) has approved Emcitate® (tiratricol) for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency. Emcitate is the first and only medicine authorised in the EU to treat MCT8 deficiency. The full indication is: Emcitate is indicated for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley Syndrome), from birth.
Nicklas Westerholm, CEO of Egetis, commented: “We are proud of the European Commission approval of Emcitate, which marks the first and only approved treatment for patients with MCT8 deficiency. This approval represents the single most important milestone in Egetis’ history and a major step forward in building a sustainable rare disease company. We are delighted to bring this much needed new treatment to patients.
“I would like to thank all patients, parents, caregivers and investigators who have taken part in the comprehensive development program for Emcitate and all Egetis employees and collaborators for their dedicated and hard work, in particular the group of Prof. Dr. Edward Visser at the Erasmus University Medical Center, Rotterdam, The Netherlands.
“We look forward to initiating pricing and reimbursement processes and discussions in Europe and expect the first launch in the second quarter of 2025.”
MCT8 deficiency is a rare, chronic and severely debilitating disease caused by mutations in the gene coding for the thyroid hormone transporter MCT8 protein. This prevents thyroid hormones from entering cells in the brain and delays brain development in individuals with MCT8 deficiency. This lack of thyroid hormone in the brain leads to severe intellectual and motor disability. Patients only rarely achieve independent sitting, and most will not be able to maintain head control.
At the same time, there is a build-up of thyroid hormones in other parts of the body, which can cause peripheral thyrotoxicosis manifesting itself in symptoms such as increased heart rate, weight loss, muscle weakness and restless sleep.
The approval of Emcitate is primarily based on Triac Trial I (clinicaltrials.gov identifier NCT02060474, Groeneweg et al. 2019), supported by The Erasmus University Medical Center Cohort Study (van Geest et al. 2022) and preliminary results of Triac Trial II (NCT0239645).
Triac Trial I was a single-arm, open-label trial conducted in children and adults treated with an individually adjusted dose of tiratricol for up to 12 months. Emcitate reduced the mean serum T3 concentration by more than 63% at month 12. All patients improved in at least one of the study endpoints: body weight, resting heart rate, or systolic blood pressure. The average number of premature atrial contractions (extra heartbeats) also decreased.
The most frequent side effects in patients treated with Emcitate were excessive sweating, irritability, anxiety and nightmares. These reactions usually occurred at the start of treatment and / or when the dose was increased, and generally resolved during treatment.
The decision from the European Commission follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on December 12, 2024. The EU approval is applicable to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein. Emcitate was granted Orphan Drug Designation (ODD) in 2017. Maintenance of ODD was recently confirmed by the Committee for Orphan Medicinal Products (COMP) of the EMA.
- Groeneweg et al. 2019 Lancet Diabetes Endocrinol. 7(9):695-706.
- van Geest et al. 2022 J Clin Endocrinol Metab. 107(3):e1136–e1147
