SEP 27 2026
Egetis Therapeutics — Emcitate (tiratricol) $EGTX
for MCT8 deficiency
About
MCT8 deficiency is a rare inherited disorder that severely disrupts brain development, leading to profound intellectual disability and major problems with speech and movement. Emcitate (tiratricol) is a thyroid hormone analog designed to deliver a form of thyroid hormone that can bypass the body's defective transport system. By providing this essential hormone directly to the brain, the treatment could potentially address the core neurological deficits of the condition.
Pivotal trial
This is a double-blind, randomized phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. Male participants, from 4 years of age (at randomization) and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching the rescue criterion (serum total triiodothyronine \[T3\] \> upper limit of normal \[ULN\] of the participant's normal range, for a ...
Source: ClinicalTrials.gov
Also in development
Drug Watch — recent news on Emcitate
- Egetis Receives Conditional FDA Acceptance of Proprietary Name Emcitate® - Finansavisen
- Egetis Announces FDA Acceptance and Priority Review of NDA for Emcitate(R) (tiratricol) for MCT8 Deficiency - ACCESS Newswire
- Egetis Eager To Launch Ultra Rare Disease Drug Emcitate - insights.citeline.com
- Emcitate Recommended for Peripheral Thyrotoxicosis in AHDS - Medscape