Tiratricol Medicine 08 June 2021 Extended indication Treatment of children and adults with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)) Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Tiratricol Domain Neurological disorders Reason of inclusion New medicine (specialité) Main indication Neurological disorders other Extended indication Treatment of children and adults with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)) Current proprietary name Already available biosimilars / generics Proprietary name Emcitate Manufacturer Rare Thyroid Therapeutics Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Thyroid hormone receptor agonists. This medicine has a similar structure to and works in the same way as the thyroid hormone T3. The difference is that, unlike T3, it can enter developing nerve cells without the MCT8 transporter protein. This is expected to allow the medicine to enter nerve cells in patients with Allan-Herndon-Dudley syndrome, replacing the hormone that they cannot transport, and thereby allowing the nerves to develop properly and relieving symptoms of the disease. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Geen. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT02396459, NCT02060474 Additional remarks All patients were treated with Triac (Téatrois tablets 350ug, Rare Thyroid Therapeutics) by individualized dose-escalation, following a pre-defined dose-escalation protocol. After the initial dose of Triac (350ug) was administered and no predefined dose-limiting toxicities were observed, the daily dose was increased progressively in 350ug steps, with a goal of attaining serum total T3 concentrations within the target range of 1·4-2·5 nmol per liter. The maintenance Triac dose was continued throughout the rest of the study period, but could be further adjusted according to the dose-escalation protocol if T3 concentrations were outside the target range during control visits. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References orpha.net, erfelijkheid.nl Additional remarks Tot op heden werden minstens 132 families met 320 getroffen individuen met het Syndroom van Allan-Herndon-Dudley gerapporteerd in de literatuur (wereldwijd). In Nederland betreft dit hooguit enkele patiënten. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References AdisInsight Additional remarks Other information Additional remarks