Tiratricol

EMCITATE is indicated for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome). EMCITATE 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.

Tiratricol is a thyroid hormone receptor (THR) agonist which, unlike triiodothyronine (T3) and thyroxine (T4), can enter cells independent of the transmembrane transporter protein MCT8. In patients with MCT8 deficiency, X-linked loss-of-function mutations in the SLC16A2 gene encoding MCT8 impair T3 entry into target tissues. This results in insufficient T3 activity in the brain concurrent with persistent peripheral thyrotoxicosis from accumulation of circulating T3. Intracellular tiratricol activity at THR-β, independent of MCT8-mediated membrane transport, can modulate hypothalamic-pituitary-thyroid axis signaling and reduce circulating T3 in patients with MCT8 deficiency.

Individualize dosage based on weight, total T3 levels, and signs and symptoms of thyrotoxicosis. Assess total T3 levels by liquid chromatography tandem mass spectrometry. EMCITATE tablets for oral suspension must be dispersed in room temperature drinking water and administered orally or by enteral feeding tube. Do not swallow tablets whole. Do not chew or crush tablets. Refer to the Full Prescribing Information for detailed dosage and administration instructions.

EMCITATE is contraindicated in patients with primary hyperthyroidism. Primary hyperthyroidism.

Boxed warning (not for treatment of obesity or for weight loss): Thyroid hormones, including EMCITATE, either alone or with other therapeutic agents, should not be used for the treatment of obesity or for weight loss. Thyrotoxicosis: Signs and symptoms of thyrotoxicosis (e.g., increased heart rate, elevated blood pressure, diarrhea, hyperhidrosis, irritability, insomnia, nightmares) have occurred with EMCITATE during treatment initiation and dose titration. Monitor 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 cause an overestimation of T3.

Most common adverse reactions (≥ 5%): diarrhea, vomiting, rash, and hyperhidrosis.

Not recommended with other thyroid medications. See full prescribing information for drugs that may interact with EMCITATE and alter the therapeutic response ( 7.2 - 7.4 ).

Store refrigerated at 2°C to 8°C (36°F to 46°F), and keep protected from light.