Egetis Announced FDA Acceptance and Priority Review of NDA for Emcitate® (tiratricol) for MCT8 Deficiency
· Egetis completed the U.S. New Drug Application (NDA) for Emcitate® in January
· NDA for Emcitate accepted for filing by the U.S. Food and Drug Administration on March 27, 2026
· Priority Review granted and PDUFA target action date set to September 28, 2026
· Egetis received Notice of Allowance for MCT8 deficiency composition patent in the U.S.
· Tiago Nunes appointed Chief Medical Officer
· Egetis successfully carried out an oversubscribed directed share issue amounting to SEK 350 million / USD 38 million (gross)
Financial overview January-March
· Quarterly revenue MSEK 13.4 (12.7), +9 % at constant exchange rates
· Quarterly loss MSEK -94.5 (-62.9)
· Cash flow for the quarter MSEK -72.6 (-74.2)
· Cash at the end of the quarter amounted to MSEK 142.5 (272.8)
· Earnings per share before/after dilution SEK -0.2 (‑0.2)
Significant events during the quarter
· Egetis completed U.S. rolling NDA submission for Emcitate® for treatment of MCT8 deficiency
· NDA submission for Emcitate® successfully validated by the FDA on March 27, 2026
· Priority Review granted and PDUFA target action date set to September 28, 2026
· FDA’s rare pediatric disease priority review program extended until 2029 – Egetis is eligible to receive a Priority Review Voucher (PRV), upon potential approval.
· Egetis received Notice of Allowance for MCT8 deficiency composition patent in the U.S.
· Egetis’ partner Fujimoto had a pre-application consultation for drugs with Japan’s PMDA. The NDA in Japan is expected to utilize existing data generated from the global clinical development program
Significant events after the quarter
· Egetis received conditional FDA acceptance of proprietary name Emcitate®
· Tiago Nunes appointed Chief Medical Officer
· Birgitte Volck and Jay Donovan Wu elected to the Board of directors at the Annual General Meeting (AGM)
· Egetis successfully carried out an oversubscribed directed share issue amounting to SEK 350 million (gross)
Comments from the CEO
Acceptance of NDA and PDUFA date
The most significant event during the first quarter of 2026 was the acceptance of our New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol) for the treatment of MCT8 deficiency. In addition, the application was granted a Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date, or FDA decision date, of September 28, 2026. The Priority Review highlights the robust dataset we have in this rare genetic disease, with the NDA based on results from Triac Trial I (46 patients), Triac Trial II (22 patients), ReTRIACt (15 patients), EMC Cohort Study (67 patients), EMC Survival Study (>600 patients) and the U.S. Expanded Access Program (approximately 40 patients included in the NDA). The U.S. market is expected to be our largest market by far, and we look forward to continuing our collaboration with the FDA during the NDA Priority Review process. Furthermore, in the Day 60 NDA acceptance letter the agency stated that it is not currently planning to hold an advisory committee meeting to discuss the application.
Priority review voucher requested
As Emcitate has been granted Rare Pediatric Disease Designation by the FDA, Egetis is eligible to receive a Priority Review Voucher (PRV), upon potential approval. In early February 2026 FDA’s Rare Pediatric Disease Priority Review program was prolonged from its previous sunset deadline of September 30, 2026 until September 30, 2029. Through the program, a company that obtains FDA approval for an eligible therapy is awarded a voucher that can be used to secure a priority review for a future NDA. Priority review shortens the FDA’s standard review period from ten months to approximately six months. PRVs are transferable and can be sold to other companies, creating an active secondary market for these vouchers. PRVs sold in 2025-2026 have fetched USD 150–200 million each. Egetis is entitled to 50 % of net proceeds from any Emcitate-related PRV sale, with the rest paid as earnout to Rare Thyroid Therapeutics International AB sellers following Emcitate’s acquisition.
Notice of Allowance for MCT8 deficiency composition patent
As previously communicated, the United States Patent and Trademark Office (USPTO) issued a Notice of Allowance for the Company’s patent application No. 19/261,360 entitled “Pharmaceutical Compositions for Treating MCT8 Deficiency”.
The allowed patent provides protection for a novel composition, which contains tiratricol as the active ingredient, designed to correct the disrupted thyroid hormone signaling characteristic of MCT8 deficiency. The allowed claims cover, among other things, a method of treating MCT8 deficiency with a pharmaceutical composition that encompasses tiratricol, including dosing regiments, and tiratricol compositions with specific excipients. This represents a significant milestone in strengthening our intellectual property portfolio. We expect the resulting patent will be Orange Book-listable, with an anticipated expiration date of 2045.
Commercialization of Emcitate in the EU
On May 1, 2025, we initiated the launch of Emcitate in the first country, Germany. All patients in our Managed Access Program in Germany had transitioned to commercial product by the third quarter of 2025. In parallel, several new MCT8 deficiency patients continue to be identified, some of whom have already started treatment with Emcitate.
In October, we initiated price negotiations within the German reimbursement process (AMNOG), and according to standard AMNOG timelines, we expect the process to conclude in Q2 2026.
The revenue for Emcitate sales in Europe during the first quarter of 2026 was MSEK 13.4, a 9% increase at constant exchange rates (CER) compared with 2025.
In Germany we continue to develop the market through meetings with physicians, congress presence, educational initiatives and disease awareness activities. We are continuing our engagements primarily with pediatric endocrinologists and neurologists. In February a web-based certified Continuous Medical Education program focused on MCT8 deficiency was launched by Egetis in Germany and in Q1 we exhibited at four scientific congresses in Germany and Austria.
In November, we initiated the pricing and reimbursement process in Italy, and during the first quarter 2026 we have received our first commercial orders from Italy through a regional agreement.
We are planning to resubmit our pricing and reimbursement dossier in France during the second quarter of 2026. Preparations are ongoing to submit a pricing and reimbursement dossier in Spain. In parallel, we have successfully implemented alternative reimbursement solutions in certain other EU countries, like Switzerland and Austria.
Pre-application consultation for drugs with Japan’s PMDA
Our Japanese partner Fujimoto Pharmaceuticals, who has an exclusive license for the development and commercialization of Emcitate for MCT8 deficiency in Japan, had a Pre-application consultation for drugs with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan regarding the regulatory pathway and data package for the marketing application of Emcitate.
As of October 2024, 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 NDA in Japan for Emcitate is expected to utilize existing data generated from the global clinical development program. Egetis and Fujimoto are evaluating the submission timelines for the NDA.
Markets Outside U.S., Europe and Japan
Our distribution partner companies Er-Kim (Turkey, Central, Eastern, and Southeastern Europe) and Taiba rare (Gulf region) are actively identifying patients in their respective territories and are now in the process of initiating funded treatment to more patients. This work generated sales revenues from named patient sales in Poland and Turkey in Q1.
Preparatory launch activities in the U.S.
With the successful progress on the regulatory front in the U.S. we have accelerated our preparatory launch activities and the build out of our commercial and medical affairs infrastructure. Our U.S. headcount was merely three employees in October last year and we are now around a dozen FTEs. We plan to be around 25 FTEs at the time of launch of Emcitate, planned for Q4 2026. We have also identified a third-party logistics provider (3PL) as well as a specialty pharmacy to assist us in providing Emcitate to patients with MCT8 deficiency after approval. As previously communicated, we recently launched updated healthcare provider (HCP) and caregiver disease education websites, at www.mct8deficiency.com and www.lifewithmct8deficiency.com respectively, designed to deliver clearer, more comprehensive, and easier‑to‑navigate information tailored to each audience.
Expanded Access Program (EAP) in the U.S.
At FDA’s request, Egetis has implemented an Expanded Access Program (EAP) in the U.S. Currently, 17 hospitals are included, and five additional hospitals are in the activation process. Currently approximately 50 patients are receiving tiratricol in the EAP. The EAP allows physicians to access tiratricol for patients not eligible for clinical trials prior to marketing approval, as well as for continued treatment of patients who completed the ReTRIACt and Triac Trial II studies.
For more information about the EAP program, see:
https://clinicaltrials.gov/study/NCT05911399
Cash
We report cash of approximately SEK 142.5 million as of March 31, 2026. Post period, on April 21, 2026, we successfully carried out an oversubscribed directed share issue amounting to SEK 350 million (approximately USD 38 million) (gross) at SEK 5.25 per share, corresponding to the closing price on Nasdaq Stockholm on April 21, 2026.
Outlook
2026 is a year marked by several important milestones for Egetis. Our team focusses on delivering three key priorities:
1. Successfully engage with FDA during the review of the Emcitate NDA to gain FDA approval.
2. Preparatory launch activities in the USA.
3. Optimize pricing- and reimbursement and continue launch of Emcitate in Europe.
Nicklas Westerholm, CEO
