Deoxythymidine / deoxycytidine Medicine 07 June 2022 Extended indication Thymidine Kinase 2 (TK2) Deficiency Therapeutic value No estimate possible yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Zogenix Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Unknown Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity New medicine with Priority Medicines (PRIME) ATMP No Submission date March 2023 Expected Registration December 2023 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Momenteel zijn er alleen open label studies gaande wat het lastig maakt om een goede therapeutische waarde in te schatten. Duration of treatment Frequency of administration Dosage per administration 400 mg/kg/day References NCT04581733 Additional remarks Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks