SEP 27 2026
Egetis Therapeutics — Emcitate (tiratricol) $EGTX
for MCT8 deficiency
About
Allan-Herndon-Dudley syndrome is a rare genetic disorder that impairs brain development, leading to intellectual disability and difficulties with speech and movement. Emcitate (tiratricol) is a synthetic thyroid hormone analog that mimics the action of thyroid hormones in the body. By restoring thyroid hormone function, it may help improve neurological symptoms in patients with this 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 Uses Pediatric Endocrine Meeting to Build Momentum for Emcitate - TipRanks
- Egetis Therapeutics AB submits rolling NDA for tiratricol - European Biotechnology Magazine
- Egetis Eager To Launch Ultra Rare Disease Drug Emcitate - Citeline News & Insights
- Emcitate Recommended for Peripheral Thyrotoxicosis in AHDS - Medscape