Egetis Therapeutics AB (publ) announced that the Company has submitted a marketing authorisation application (MAA) to the European Medicines Agency (EMA) for Emcitate (tiratricol) for the treatment of MCT8 deficiency. This is an important step towards bringing the first approved treatment for MCT8 deficiency to patients and a transformative milestone for the Company. Emcitate has been granted Orphan Drug Designation by the EMA for MCT8 deficiency, and will following approval by the European Commission be eligible for 10 years of market exclusivity within the EU.

The median review time for marketing authorisation applications in the EU is around 13-14 months. As agreed with the US FDA, Egetis is conducting a randomized, placebo-controlled pivotal study (ReTRIACt) in 16 evaluable patients to verify the results on thyroid hormone T3 levels seen in previous clinical trials and publications. The study started recruiting in July this year and patient recruitment is proceeding according to plan.

Topline results are expected during the first half of 2024 and Egetis intends to submit a new drug application (NDA) in the USA for Emcitate in mid-2024 under the Fast-Track Designation granted by the FDA. In addition to the completed and ongoing clinical trials with Emcitate, more than 180 patients in over 25 countries, are currently being treated with Emcitate as part of the Company's Expanded Access/Named Patient Use/Compassionate Use programs.