Emcitate is under development for the treatment of patients with MCT8 deficiency, a highly debilitating rare disease with no available treatment. In previous studies (Triac Trial I and a long-term real-life study) Emcitate has shown highly significant and clinically relevant results on serum thyroid hormone T3 levels and secondary clinical endpoints. As a result of regulatory interaction Egetis intends to submit a marketing authorisation application (MAA) for Emcitate to the European Medicines Agency (EMA) in the early autumn of 2023 based on existing clinical data.
In the US, after discussions with the FDA, Egetis will conduct a small randomized, placebo-controlled study in 16 patients to verify the results on T3 levels seen in previous clinical trials and publications. Egetis intends to submit a new drug application (NDA) in the US for Emcitate in the fourth quarter of 2023 under the Fast-Track Designation granted by FDA.
Emcitate holds Orphan Drug Designation (ODD) for MCT8 deficiency and resistance to thyroid hormone type beta (RTH- β) in the US and the EU. Emcitate has been granted Rare Pediatric Disease Designation (RPD) which gives Egetis the opportunity to receive a Priority Review Voucher (PRV) in the US, after approval.
Aladote® a first-in-class drug candidate, is being developed to reduce the risk of acute liver injury associated with acetaminophen/paracetamol poisoning. A proof of principle study has been successfully completed and the design of the next study is being finalised. Aladote® has been granted Orphan Drug Designation in the US. Design of pivotal Phase IIb/III study for Aladote® finalized after completed interactions with FDA, EMA and MHRA