Stockholm, Sweden - The European Commission (EC) has approved Emcitate® (tiratricol) for the treatment of patients with monocarboxylate transporter 8 (MCT8) deficiency, marking a significant milestone as the first and only authorized medicine in the EU for this rare condition. Egetis Therapeutics AB (publ) (Nasdaq Stockholm: EGTX) made the announcement, heralding a new era for MCT8 deficiency treatment.
The approval is specifically for the treatment of peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley Syndrome, from birth. This decision follows a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on December 12, 2024.
Clinical Evidence Supporting Approval
The approval of Emcitate is primarily based on the results of Triac Trial I (clinicaltrials.gov identifier NCT02060474), a single-arm, open-label trial. The study, led by Prof. Dr. Edward Visser at the Erasmus University Medical Center, Rotterdam, involved children and adults treated with individually adjusted doses of tiratricol for up to 12 months. Key findings included:
- A reduction of more than 63% in mean serum T3 concentration at month 12.
- Improvements in at least one of the study endpoints for all patients, including body weight, resting heart rate, or systolic blood pressure.
- A decrease in the average number of premature atrial contractions.
The Triac Trial I data was supported by The Erasmus University Medical Center Cohort Study (van Geest et al. 2022) and preliminary results of Triac Trial II (NCT0239645).
Management Commentary
Nicklas Westerholm, CEO of Egetis, expressed pride in the EC's decision, stating, "We are proud of the European Commission approval of Emcitate, which marks the first and only approved treatment for patients with MCT8 deficiency...We are delighted to bring this much needed new treatment to patients."
Westerholm also extended gratitude to the patients, parents, caregivers, investigators, and Egetis employees who contributed to the comprehensive development program for Emcitate.
MCT8 Deficiency: An Unmet Medical Need
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 genetic defect impairs the transport of thyroid hormones into brain cells, leading to developmental delays and intellectual and motor disabilities. Patients often struggle to achieve independent sitting and head control.
Furthermore, the buildup of thyroid hormones in other parts of the body results in peripheral thyrotoxicosis, manifesting as increased heart rate, weight loss, muscle weakness, and restless sleep.
Side Effects and Regulatory Status
The most frequent side effects observed in patients treated with Emcitate were excessive sweating, irritability, anxiety, and nightmares. These reactions typically occurred at the start of treatment or when the dose was increased and generally resolved during treatment.
Emcitate received Orphan Drug Designation (ODD) in 2017, which was recently confirmed by the Committee for Orphan Medicinal Products (COMP) of the EMA. The EU approval extends to all 27 European Union member states, as well as Iceland, Norway, and Liechtenstein.
Future Plans
Egetis Therapeutics is now focused on initiating pricing and reimbursement processes and discussions across Europe. The company anticipates the first launch of Emcitate in the second quarter of 2025.
