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Deoxythymidine / deoxycytidine

Extended indication Thymidine Kinase 2 (TK2) Deficiency
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Deoxythymidine / deoxycytidine
Domain Neurological disorders
Reason of inclusion New medicine (specialité)
Main indication Muscular diseases other
Extended indication Thymidine Kinase 2 (TK2) Deficiency
Manufacturer UCB
Portfolio holder UCB
Route of administration Oral
Budgetting framework Extramural (GVS)
Centre of expertise onbekend
Additional remarks werkingsmechanisme: 'restoring mitochondrial DNA (mtDNA) replication fidelity' (bron UCB pijplijn).

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
Particularity New medicine with Priority Medicines (PRIME)
ATMP No
Submission date 2024
Expected Registration March 2025
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Ultra weesgeneesmiddel. Fabrikant verwacht registratie in maart 2025.

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation Momenteel zijn er alleen open label studies gaande wat het lastig maakt om een goede therapeutische waarde in te schatten.
Frequency of administration 1 times a day
Dosage per administration 400 mg/kg/day
References NCT04581733
Additional remarks Dosering: 'All participants will receive MT1621 up to a target dose of 400mg/kg/day' (NCT04581733)

Expected patient volume per year

Patient volume

< 10

Market share is generally not included unless otherwise stated.
References TK2-Related Mitochondrial DNA Maintenance Defect, Myopathic Form. Julia Wang. 2012 (1); expertopinie (2)
Additional remarks Een zeer zeldzame ziekte, 107 gevallen bekend wereldwijd (1). In Nederland is de inschatting dat er maximaal 10 patiënten zouden kunnen zijn maar waarschijnlijk minder (2). Aanvulling: zover bekend twee patiënten in Nederland

Expected cost per patient per year

Additional remarks onbekend

Potential total cost per year

Additional remarks onbekend

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No

Other information

There is currently no futher information available.