Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate® (tiratricol) for MCT8 Deficiency
March 27, 2026
- NDA submission for Emcitate® successfully validated by the FDA
- Priority Review granted and PDUFA target action date set to September 28, 2026
- Emcitate® (tiratricol) is eligible to receive a Priority Review Voucher (PRV) upon approval
- If approved, Emcitate® (tiratricol) would become the first approved treatment in the United States for MCT8 deficiency
Stockholm, Sweden, March 27, 2026. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. Egetis completed the rolling NDA in January 2026. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026.
Nicklas Westerholm, CEO of Egetis, commented: “MCT8 deficiency is a rare, devastating, life-shortening disorder with no approved treatment in the U.S. The acceptance of our NDA with Priority Review represents an important step in the path to making Emcitate® (tiratricol) available to MCT8 deficiency patients in the U.S. The Priority Review also highlights the robust dataset we have in this rare genetic disease, with the NDA based on results from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the U.S. Expanded Access Program. We look forward to collaborating with the FDA during the NDA Priority Review process and will in parallel continue to build our medical affairs and market access capabilities towards a potential U.S. launch in the fourth quarter of this year.”
A Priority Review will direct overall attention and resources to the evaluation of applications for drugs that, if approved, would represent significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. A Priority Review means FDA’s goal is to take action on an application within 6 months, compared to 10 months under standard review.
Emcitate® (tiratricol) has previously been granted Rare Pediatric Disease Designation by the FDA and is eligible to receive a Priority Review Voucher (PRV) upon approval, and a PRV was consequently requested in connection with the submission. The transferability of PRVs has created an active secondary market for these vouchers, selling for $150-$205 million per voucher in 2025/26.
