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Doxecitine / doxiribtimine

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

Product

Active substance Doxecitine / doxiribtimine
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
Mechanism of action Other
Route of administration Oral
Therapeutical formulation Solution for parenteral / oral use
Centre of expertise Radboud UMC
Additional remarks Werkingsmechanisme: 'restoring mitochondrial DNA (mtDNA) replication fidelity' (bron UCB pijplijn).

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Particularity New medicine with Priority Medicines (PRIME)
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Ultra weesgeneesmiddel. Indieningsdatum en verwachte registratie op basis van informatie fabrikant.

Therapeutic value

Current treatment options 'best supportive care'
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 3 times a day
Dosage per administration Start dosering: 260 mg/kg/dag tot maximaal 800mg/kg/dag
References NCT04581733 / NCT05017818 / NCT03701568 / NCT06590493. UCB data on file

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.