Timrepigene emparvovec

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

Product

Active substance	Tiratricol
Domain	Neurological disorders
Reason of inclusion	Generic
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.
Proprietary name	Emcitate
Manufacturer	Rare Thyroid Therapeutics
Mechanism of action	Receptor agonist
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
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 route	Centralised (EMA)
ATMP	No
Submission date	2025
Expected Registration	2026
Orphan drug	Yes
Registration phase	Clinical trials
Additional remarks	IHSI tijdslijnen - 24 maanden vanaf het eerste kwartaal van 2024.

Current treatment options	Geen.
Therapeutic value	No estimate possible yet
Duration of treatment	continuous
Frequency of administration	1 times a day
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.

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.

Horizonscan vermeldt geen prijsinformatie van generieke geneesmiddelen of biosimilars om het eventueel beïnvloeden van de markt te voorkomen.

Horizonscan vermeldt geen prijsinformatie van generieke geneesmiddelen of biosimilars om het eventueel beïnvloeden van de markt te voorkomen.

There is currently nothing known about off label use.

Indication extension	No
References	AdisInsight

There is currently no futher information available.