Extended indication Treatment of children and adults with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Tiratricol
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Other neurological disorders
Extended indication Treatment of children and adults with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS))
Proprietary name Emcitate
Manufacturer Rare Thyroid Therapeutics
Mechanism of action Receptor agonist
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)
Additional comments 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)
ATMP No
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options Geen.
Therapeutic value No estimate possible yet
Duration of treatment continuous
Frequency of administration 1 times a day
References NCT02396459, NCT02060474
Additional comments 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

References orpha.net, erfelijkheid.nl
Additional comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References AdisInsight

Other information

There is currently no futher information available.