Summary: development and regulatory status as of September 2026

The FDA approved tiratricol (Emcitate) tablets for oral suspension on 28 September 2026 for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency (Allan-Herndon-Dudley syndrome), with a limitation of use stating that it is not recommended for the treatment of primary hypothyroidism. The approval was granted to Egetis Therapeutics US Inc., and it is the first FDA-approved treatment for MCT8 deficiency. Egetis completed a rolling NDA submission on 29 January 2026, and on 27 March 2026 announced that the FDA had accepted the application with priority review and a PDUFA target action date of 28 September 2026. Tiratricol received orphan drug designation in January 2019, rare pediatric disease designation in November 2020, fast track designation in 2021, and breakthrough therapy designation in 2025, and the FDA granted Egetis a rare pediatric disease priority review voucher in connection with the approval. The prescribing information states that efficacy was evaluated in two studies (NCT05579327 and NCT02060474); Egetis states that the approval was also supported by Triac Trial II, the EMC Cohort Study, the EMC Survival Study, and the U.S. Expanded Access Program. Egetis expects commercial availability in the United States eight to ten weeks after approval. In the European Union, tiratricol was authorised on 12 February 2025 as a hybrid medicine whose reference product is Téatrois, a 0.35 mg tablet authorised in France in 1974, and it was launched in Germany on 1 May 2025. A U.S. composition patent was granted on 5 May 2026, and Egetis expects it to expire in 2045. In Japan, the licensee Fujimoto Pharmaceuticals expects to submit an NDA at the beginning of 2027.

Regulatory submission: NDA submitted January 2026 and approved September 2026

“Stockholm, Sweden, January 29, 2026.” (opens the source at this quote in a new tab)
“Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the Company has completed the rolling New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for Emcitate® (tiratricol) for treatment of patients with MCT8 deficiency.” (opens the source at this quote in a new tab)

“Egetis completed the rolling NDA in January 2026.” (opens the source at this quote in a new tab)

“For Immediate Release: September 28, 2026” (opens the source at this quote in a new tab)
“The U.S. Food and Drug Administration today approved Emcitate (tiratricol) tablets for oral suspension to treat peripheral thyrotoxicosis (excess thyroid hormone levels in the blood that causes symptoms, such as rapid heart rate, increased blood pressure and adverse effects on metabolism) in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome.” (opens the source at this quote in a new tab)

Launch: Anticipated eight to ten weeks after the September 2026 approval

“STOCKHOLM, SWEDEN, September 28, 2026.” (opens the source at this quote in a new tab)
“The Company expects EMCITATE to be commercially available in the United States in eight to ten weeks post approval.” (opens the source at this quote in a new tab)

FDA approval on 28 September 2026

“Emcitate is the first therapy approved by the FDA to treat symptoms of this very rare, devastating genetic disease.” (opens the source at this quote in a new tab)
“The approval of Emcitate was granted to Egetis Therapeutics US Inc.” (opens the source at this quote in a new tab)

Drug name	Active ingredient	Approval date	FDA-approved use on approval date
“Emcitate”	“tiratricol”	“9/28/2026”	“To treat peripheral thyrotoxicosis in patients with MCT8 deficiency”

“Initial U.S. Approval: 2026” (opens the prescribing information at this quote)
“EMCITATE is indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome).” (opens the prescribing information at this quote)

Evidence supporting approval

“The efficacy of EMCITATE in patients with MCT8 deficiency was evaluated in two studies; Study 1 (NCT05579327) and Study 2 (NCT02060474).” (opens the prescribing information at this quote)

“The FDA approval of EMCITATE was supported by a comprehensive clinical development program evaluating EMCITATE in patients with MCT8 deficiency, including ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center (EMC) Cohort Study, EMC Survival Study and the U.S. Expanded Access Program.” (opens the source at this quote in a new tab)

“The effectiveness of Emcitate was evaluated in two clinical studies in patients ranging in age from infants to adults, including an international, multi-center, randomized, placebo-controlled trial (NCT05579327) and a longer-term open-label study.” (opens the source at this quote in a new tab)
“Across both studies, patients treated with Emcitate had reductions in excess thyroid hormone levels in the bloodstream and improvements in cardiovascular and metabolic symptoms impacted by thyroid levels, such as systolic blood pressure and heart rate.” (opens the source at this quote in a new tab)

Expedited programs: priority review and special FDA designations

“Emcitate was granted Orphan Drug, Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations as well as Priority Review.” (opens the source at this quote in a new tab)

“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.” (opens the source at this quote in a new tab)

Rare pediatric disease priority review voucher

“In connection with the approval of EMCITATE, the FDA granted Egetis a Rare Pediatric Disease Priority Review Voucher (PRV).” (opens the source at this quote in a new tab)
“The Company currently expects to explore monetization of the PRV, which could potentially occur in the fourth quarter of 2026, subject to market conditions.” (opens the source at this quote in a new tab)

“Egetis is entitled to 50 percent of net proceeds from any Emcitate-related PRV sale, with the rest paid as earnout to Rare Thyroid Therapeutics International AB sellers following acquisition of Emcitate in 2020.” (opens the source at this quote in a new tab)

Advisory committee

“Thus far, the review has been constructive, and the mid-cycle as well as the late-cycle meetings have been held with the agency, which has confirmed that FDA expects to finish its review by the PDUFA date of September 28, 2026 and does not plan to hold an advisory committee meeting.” (opens the source at this quote in a new tab)

Special FDA designations: orphan drug designation

Field	Quoted record
Generic name	“tiratricol”
Date designated	“01/16/2019”
Orphan designation	“Treatment of Monocarboxylate 8 Transporter (MCT8) Deficiency (Allan-Herndon-Dudley-Syndrome)”
FDA orphan approval status	“Not FDA Approved for Orphan Indication”
Sponsor	“Rare Thyroid Therapeutics”

Special FDA designations: Fast Track

“October 8, 2021” (opens the source at this quote in a new tab)
“Egetis Therapeutics AB (publ) (ticker: EGTX) today announced that the U.S. Food and Drug Administration (FDA) has granted the company’s lead candidate drug Emcitate®, currently in a pivotal study, Fast Track Designation for the treatment of the rare genetic disease MCT8 deficiency.” (opens the source at this quote in a new tab)

Special FDA designations: Breakthrough Therapy

“Stockholm, Sweden, July 15, 2025.” (opens the source at this quote in a new tab)
“Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (NASDAQ Stockholm: EGTX), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for tiratricol, as a treatment for monocarboxylate transporter 8 (MCT8) deficiency.” (opens the source at this quote in a new tab)

Special FDA designations: Rare Pediatric Disease

“Emcitate holds Orphan Drug Designation (ODD) in both EU and the US and was granted US Rare Pediatric Disease Designation (RPD) in November 2020 and in October 2021 the product received Fast Track status from the FDA.” (opens the source at this quote in a new tab)

Patent protection: U.S. composition patent

“On May 5, 2026, the United States Patent and Trademark Office (USPTO) granted Patent No. US 12611383B1 for the Company’s patent application No. 19/261,360 entitled “Pharmaceutical Compositions for Treating MCT8 Deficiency”.” (opens the source at this quote in a new tab)
“The claims cover, among other things, a method of treating MCT8 deficiency with the claimed pharmaceutical composition that encompasses tiratricol, dosing regimens, and tiratricol compositions with specific excipients.” (opens the source at this quote in a new tab)
“Egetis expects the granted patent will be Orange Book-listable, with a patent expiration date in 2045.” (opens the source at this quote in a new tab)

Approval and launch outside the United States: European Union authorisation

“Emcitate received a marketing authorisation valid throughout the EU on 12/02/2025.” (opens the source at this quote in a new tab)
“Emcitate contains the active substance tiratricol and is a hybrid medicine.” (opens the source at this quote in a new tab)
“The reference medicine for Emcitate is Téatrois, which was marketed in France for the treatment of thyroid hormone resistance syndrome.” (opens the source at this quote in a new tab)
“MCT8 deficiency is rare, and Emcitate was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 November 2017.” (opens the source at this quote in a new tab)
“A study involving patients with MCT8 deficiency has shown that Emcitate reduces the average levels of the thyroid hormone T3 and leads to small improvements in signs of peripheral thyrotoxicosis.” (opens the source at this quote in a new tab)

“EMA has recommended granting a marketing authorisation in the European Union (EU) for Emcitate (tiratricol), an oral treatment for peripheral thyrotoxicosis (inappropriately high levels of circulating thyroid hormones) in patients with monocarboxylate transporter 8 (MCT8) deficiency.” (opens the source at this quote in a new tab)

Approval and launch outside the United States: European Union application and legal basis

“The applicant Rare Thyroid Therapeutics International AB submitted on 6 October 2023 an application for marketing authorisation to the European Medicines Agency (EMA) for Emcitate, through the centralised procedure under Article 3(1) and point 4 of Annex of Regulation (EC) No 726/2004.” (opens the source at this quote in a new tab)
“The applicant initially applied for the following indication:” (opens the source at this quote in a new tab)
“Emcitate is indicated for the treatment of monocarboxylate transporter 8 (MCT8) deficiency in all age groups.” (opens the source at this quote in a new tab)
“The final applied indication was as follows:” (opens the source at this quote in a new tab)
“Emcitate is indicated for the treatment of peripheral thyrotoxicosis in patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley Syndrome), from birth.” (opens the source at this quote in a new tab)

Field	Quoted record
Legal basis	“Hybrid application (Article 10(3) of Directive No 2001/83/EC).”
Reference product	“Téatrois 0.35 mg tablet”
Reference product date of authorisation	“25-06-1974”
Reference product Member State	“France”

Approval and launch outside the United States: launch in Germany

“Egetis initiated the launch of Emcitate® in Germany on May 1, 2025.” (opens the source at this quote in a new tab)

“We initiated the launch of Emcitate in the first country, Germany, in May 2025, and in October 2025 we started price negotiations with the German national statutory health insurers, GKV-Spitzenverband.” (opens the source at this quote in a new tab)

Approval and launch outside the United States: other European countries and Japan

“In Spain we have recently submitted a national pricing and reimbursement dossier for Emcitate.” (opens the source at this quote in a new tab)
“In Italy and France, we plan to strengthen our value dossiers with survival data for Emcitate, once it has been published in a peer-reviewed journal by Erasmus Medical Center.” (opens the source at this quote in a new tab)
“Our Japanese partner Fujimoto Pharmaceuticals, who has an exclusive license for the development and commercialization of Emcitate for MCT8 deficiency in Japan, continues to prepare the Japanese NDA submission dossier, utilizing existing data generated from the global clinical development program.” (opens the source at this quote in a new tab)
“The NDA submission in Japan for Emcitate is expected to be submitted in the beginning of 2027.” (opens the source at this quote in a new tab)

Tiratricol development and approval review

“Tiratricol received its first approval on 12 February 2025 in the European Union, for the treatment of peripheral thyrotoxicosis in patients with MCT8 deficiency (Allan-Herndon-Dudley Syndrome), from birth.” (opens the source at this quote in a new tab)
“This article summarizes the milestones in the development of tiratricol leading to this first approval for MCT8 deficiency.” (opens the source at this quote in a new tab)