- EMCITATE is the first and only FDA-approved treatment for peripheral thyrotoxicosis in adults and pediatric patients with MCT8 deficiency in the United States
- Egetis RareLink™, the dedicated patient support program, provides access assistance, education and care coordination in partnership with PANTHERx® Rare Pharmacy
Stockholm, Sweden, October 27, 2026 – Egetis Therapeutics AB (publ) (“Egetis” or the “Company”) (Nasdaq Stockholm: EGTX), a commercial-stage pharmaceutical company focused on developing and delivering innovative therapies for patients with rare diseases, today announced the commercial availability of EMCITATE® (tiratricol), ahead of anticipated timelines following U.S. Food and Drug Administration (FDA) approval on September 28, 2026.
EMCITATE is indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency, also known as Allan-Herndon-Dudley syndrome. It is the first and only FDA-approved treatment for MCT8 deficiency in the United States. EMCITATE is not recommended for the treatment of primary hypothyroidism.
“Launching EMCITATE in the United States ahead of our anticipated timelines marks an important milestone for people living with MCT8 deficiency, their families and everyone who has worked toward bringing an approved treatment to this community,” said Nicklas Westerholm, chief executive officer of Egetis Therapeutics. “Following FDA approval, our focus is now on building on this momentum and helping eligible patients access treatment as quickly as possible. We remain deeply committed to the MCT8 deficiency community and grateful to the patients, families, clinicians, investigators and advocacy organizations that continue to support these efforts.”
MCT8 deficiency is a rare, devastating, life-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2 gene. These mutations impair the function of MCT8, a critical cell-surface thyroid hormone transporter responsible for thyroid hormone transport in specific cells, including in the brain. The resulting disruption of thyroid hormone signaling leads to severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis. Patients with MCT8 deficiency have a reported median life expectancy of approximately 35 years.
To support the U.S. launch, Egetis has established Egetis RareLink, its dedicated patient support program, in partnership with PANTHERx Rare Pharmacy. The program is designed to help eligible patients, caregivers and health care professionals navigate medication access, education, care coordination and ongoing treatment services.
Through Egetis RareLink, the Company aims to provide dedicated support and help eligible patients access EMCITATE. Patients and caregivers can learn more by calling 1-844-4EGETIS (1-844-434-3847) or go to EMCITATE.com.
About EMCITATE®
EMCITATE (tiratricol) is a thyroid hormone receptor agonist indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley syndrome).
Limitation of use: EMCITATE is not recommended for the treatment of primary hypothyroidism.
About MCT8 Deficiency
Monocarboxylate transporter 8 (MCT8) deficiency, also known as Allan–Herndon–Dudley syndrome (AHDS), is a rare, devastating, life-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2 gene. These mutations impair the function of MCT8, a critical cell-surface thyroid hormone transporter responsible for thyroid hormone transport in specific cells, including in the brain. Patients experience insufficient thyroid hormone activity in the central nervous system and excessive exposure to the active thyroid hormone triiodothyronine (T3) in peripheral tissues. This results in severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis.
Elevated T3 concentrations in peripheral tissues can result in a chronic hypermetabolic state affecting multiple organs, including the heart, muscles, liver and kidneys. Clinical manifestations may include failure to thrive, cardiovascular strain, muscle wasting, metabolic imbalance and increased susceptibility to infections. These systemic consequences are believed to contribute significantly to the increased morbidity and premature mortality associated with the disorder. Patients with MCT8 deficiency have a reported median life expectancy of approximately 35 years.
For more information about MCT8 deficiency, visit MCT8deficiency.com or LifewithMCT8deficiency.com.
Important Safety Information
BOXED WARNING: NOT FOR TREATMENT OF OBESITY OR FOR WEIGHT LOSS
Contraindications: EMCITATE is contraindicated in patients with primary hyperthyroidism.
Warnings and precautions
- Thyrotoxicosis: Signs and symptoms of thyrotoxicosis, including increased heart rate, elevated blood pressure, diarrhea, hyperhidrosis, irritability, insomnia and nightmares, have occurred during treatment initiation and dose titration. Monitor patients and adjust the EMCITATE dose as indicated.
- Laboratory test interference for T3 measurement: Tiratricol can cross-react with immunoassays for T3, leading to unreliable T3 results and overestimation of T3.
Adverse reactions: The most common adverse reactions (≥5%) are diarrhea, vomiting, rash and hyperhidrosis. To report suspected adverse reactions, contact Egetis Therapeutics US Inc. at 1-844-4EGETIS (1-844-434-3847) or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see the full Prescribing Information: https://www.egetis.com/wp-content/uploads/emcitate-full-prescribing-information.pdf
About Egetis Therapeutics
Egetis Therapeutics is a commercial-stage pharmaceutical company focused on developing and delivering innovative therapies for patients with rare diseases with significant unmet medical need.
Combining scientific and clinical expertise with integrated late-stage development, regulatory and commercial capabilities, and manufacturing expertise, Egetis is committed to delivering treatments and dedicated support to underserved patient communities. The Company’s lead product, EMCITATE® (tiratricol), is the first and only approved treatment for MCT8 deficiency, a rare and life-shortening genetic disorder, with marketing authorizations in the European Union and the United States.
Headquartered in Stockholm, Sweden, with operations in the U.S. and Europe, Egetis is building a sustainable global rare disease company.
For more information, visit egetis.com and follow the Company on LinkedIn.
About PANTHERx Rare
PANTHERx Rare makes rare disease care more personalized and less overwhelming by focusing on each patient and each therapy. PANTHERx experts develop relationships with patients, prescribers and pharmaceutical partners, serving as trusted advocates to support collaboration and care. Founded in Pittsburgh, Pennsylvania, in 2011, PANTHERx has grown into the largest independent rare pharmacy in the U.S. PANTHERx is licensed in all 50 states and U.S. territories and was the first national pharmacy to achieve dual rare disease accreditations from the Accreditation Commission for Health Care (ACHC) and the Utilization Review Accreditation Commission (URAC). PANTHERx received the 2026 MMIT Patient Choice Award for patient satisfaction, marking its ninth win.
For more information, visit www.pantherxrare.com.
For further information, please contact:
Nicklas Westerholm, CEO
+46 (0) 733 542 062
[email protected]
Karl Hård, Head of Investor Relations, Communications & Business Development
+46 (0) 733 011 944
[email protected]
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws, including statements regarding the commercialization and availability of EMCITATE® (tiratricol) in the United States, the timing and outcome of potential monetization of the Rare Pediatric Disease Priority Review Voucher, the expected capabilities of the Company’s patient access and distribution infrastructure, the anticipated benefits of EMCITATE for patients with MCT8 deficiency, and the Company’s broader strategic plans for the development and commercialization of therapies for rare diseases. Forward-looking statements can generally be identified by words such as “expects,” “anticipates,” “intends,” “believes,” “estimates,” “plans,” “will,” “may,” “could,” “potential,” or similar expressions, although not all forward-looking statements contain these words.
These statements are based on the Company’s current expectations, assumptions, and assessments as of the date of this press release and are subject to risks, uncertainties, and other factors that could cause actual results to differ materially from those expressed or implied. Such factors include, but are not limited to: risks related to the commercial launch and market acceptance of EMCITATE in the United States; the ability to establish and maintain adequate commercial infrastructure, including distribution, patient support, and reimbursement arrangements; the timing and terms of any monetization of the Priority Review Voucher, which is subject to market conditions and the availability of interested purchasers; potential post-marketing requirements or restrictions imposed by the FDA; the ability to maintain regulatory approvals in the United States and the European Union; competition from existing or future therapies; the Company’s ability to secure adequate funding for its operations and commercial activities; and general economic, market, and business conditions.
For a further description of risks and uncertainties that could affect the Company’s business and results, reference is made to the Company’s most recent annual report and other filings with relevant regulatory authorities available at www.egetis.com. The Company undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by applicable law or regulation.
