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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

Zogenix
Route of administration

Oral
Budgetting framework

Extramural (GVS)

Registration

Registration route

Centralised (EMA)
Type of trajectory

Accelerated assessment
Particularity

New medicine with Priority Medicines (PRIME)
ATMP

No
Submission date

August 2023
Expected Registration

May 2024
Orphan drug

Yes
Registration phase

Clinical trials

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.
Dosage per administration

400 mg/kg/day
References 
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).

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

Other information

There is currently no futher information available.