Stockholm, Sweden, December 19, 2025. Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the Company has initiated a rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol), its investigational drug for the treatment of MCT8 deficiency.
Emcitate® (tiratricol) was awarded Breakthrough Therapy Designation by the FDA in July 2025 (link to press release) and in October 2025 FDA granted a rolling NDA submission and review (link to press release) based on currently available data. In November, the Company presented positive topline results from its randomized controlled withdrawal study ReTRIACt (link to press release), representing the final piece in the clinical data package to be submitted. Consequently, the Company has now submitted the non-clinical and quality (chemistry, manufacturing and controls, or CMC) sections of the NDA to the FDA.
A rolling NDA allows completed sections of an NDA to be submitted and reviewed by the FDA on an ongoing basis, enabling earlier engagement with the FDA and supporting a more efficient review process.
In parallel Egetis is finalizing the clinical sections in the NDA to encompass the latest data and plans to complete the NDA submission early 2026, together with a request for Priority Review and thus an anticipated FDA approval in the third quarter of 2026.
Nicklas Westerholm, CEO of Egetis, commented: “We are delighted to have initiated our rolling NDA for Emcitate® (tiratricol) with the FDA. The submission of the non-clinical and CMC sections of the NDA for Emcitate® (tiratricol) marks a significant milestone for Egetis and underscores our dedication to advancing Emcitate® (tiratricol) for the benefit of patients with MCT8 deficiency.
“In parallel with the regulatory submission, Egetis is accelerating the build-out of its U.S. organization to support a potential commercial launch. The Company is establishing core U.S. capabilities across market access, medical affairs, and commercial operations to ensure launch readiness and appropriate engagement with U.S. stakeholders, including payers, healthcare providers, and patient advocacy organizations.”
At the successful pre-NDA meeting on October 21, FDA confirmed that Egetis may submit the NDA for Emcitate® (tiratricol) based on currently available data and granted a rolling NDA submission and review. The NDA will be based on results from Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study, EMC Survival Study and the U.S. Expanded Access Program.
Emcitate® (tiratricol) has received multiple FDA designations including Orphan Drug, Fast Track and Breakthrough Therapy, reflecting its potential to address serious conditions with significant unmet medical need. Emcitate® (tiratricol) has also received Rare Pediatric Disease Designation and will be eligible for a Priority Review Voucher upon NDA approval, provided it occurs before October 1, 2026, or later if the U.S. Government prolongs the PRV program by enacting the Give Kids a Chance Act of 2025 (bill S.932).
