Egetis Therapeutics Reports Progress on Emcitate for MCT8 Deficiency

Egetis Therapeutics has published its 2025 Annual Report and provided updates on the development of its lead drug candidate Emcitate® (tiratricol) for monocarboxylate transporter 8 (MCT8) deficiency. The company completed a rolling New Drug Application for Emcitate® in the USA on January 29, 2026, and the FDA is expected to confirm within 60 days that the submission is complete.

In February 2025, the European Commission approved Emcitate® as the first and only treatment for MCT8 deficiency in the EU. Egetis initiated the launch of Emcitate® in Germany on May 1, 2025. The drug is not yet approved in the USA.

Tiratricol holds Orphan Drug Designation for MCT8 deficiency and resistance to thyroid hormone beta in both the US and the EU. The active ingredient has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the FDA, which gives Egetis the opportunity to receive a Priority Review Voucher in the US after approval. As a designated Fast Track and Breakthrough Therapy, Egetis has requested Priority Review, and if granted, the FDA review should be completed within six months following the 60-day filing review period.

Based on feedback from the FDA, the NDA for Emcitate® for treatment of MCT8 deficiency will be based on currently available clinical data from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the US Expanded Access Program.

In Japan, Egetis' partner Fujimoto Pharmaceuticals recently had a Pre-application consultation with Japan's Pharmaceuticals and Medical Devices Agency regarding the regulatory pathway and data package for the marketing application of Emcitate®. New guidelines from Japan's Ministry of Health, Labour and Welfare allow for approval without Japanese patient clinical data for ultra-rare diseases where conducting clinical trials in Japan is impracticable, provided that global trial data is robust and the benefit-risk ratio is favorable. The New Drug Application in Japan for Emcitate® is expected to utilize existing data generated from the global clinical development program.

The company's other drug candidate, Aladote® (calmangafodipir), is a first-in-class drug candidate developed to reduce the risk of acute liver injury associated with paracetamol (acetaminophen) overdose. A proof of principle study has been successfully completed, and the design of a pivotal Phase IIb/III study (Albatross) has been finalized following interactions with the FDA, EMA and MHRA. The development program for Aladote® has been parked until Emcitate® marketing authorization submissions for MCT8 deficiency have been completed. Aladote® has been granted Orphan Drug Designation in both the US and the EU.