Egetis Therapeutics Receives FDA Approval for EMCITATE Treatment

Egetis Therapeutics, a commercial-stage pharmaceutical company, has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) approving EMCITATE® (tiratricol) for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency. This approval marks the first and only FDA-approved treatment for this rare genetic disorder in the United States.

MCT8 deficiency, also known as Allan, Herndon, Dudley syndrome (AHDS), is a rare, devastating, and 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 the brain. As a result, patients experience disrupted thyroid hormone signaling, characterized by insufficient thyroid hormone activity in the central nervous system and excessive exposure to the active thyroid hormone triiodothyronine (T3) in peripheral tissues. This leads to a complex disorder characterized by severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis.

The FDA approval of EMCITATE was supported by a comprehensive clinical development program, including the ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center (EMC) Cohort Study, EMC Survival Study, and the U.S. Expanded Access Program.

Egetis is committed to helping eligible patients gain timely access to EMCITATE. The Company expects EMCITATE to be commercially available in the United States in eight to ten weeks post-approval. Through Egetis RareLink, its dedicated patient support program, Egetis has established the access infrastructure, including specialty distribution and dedicated support resources, to help ensure a seamless experience for patients, caregivers, and healthcare professionals from day one. As part of its U.S. commercialization strategy, Egetis has partnered with PANTHERx® Rare to support medication access, education, care coordination, and ongoing treatment services.

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

Egetis will host a conference call and webcast for analysts and investors to discuss the FDA approval of EMCITATE beginning at 8:00 am CEST (2:00 am EDT) on Tuesday, September 29, 2026. Webcast link: live.events.inderes.com/fda-approval-sep-2026