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 Generic G 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 Dit betreft een hybride geneesmiddel. Het referentie product is niet meer op de markt. 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 agonist. 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 2025 Expected Registration 2026 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks IHSI tijdslijnen - 24 maanden vanaf het eerste kwartaal van 2024. 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 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10938520/pdf/JCRPE-16-1.pdf Additional remarks De Triac I trial toont normalisatie van T3 waarden. De resultaten van de Triac Trial II (NCT02396459) waarbij de neurologische ontwikkeling van jonge kinderen met MCT8 deficiëntie wordt onderzocht, worden verwacht in 2024 of 2025. 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