Vonicog alfa Medicine 03 June 2025 Extended indication Treatment of haemorrhage in children (aged less than 18 years) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Vonicog alfa Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Hemostasis promoting medication Extended indication Treatment of haemorrhage in children (aged less than 18 years) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or contraindicated. Current proprietary name Already available biosimilars / generics Proprietary name Veyvondi Manufacturer Shire Portfolio holder Shire Mechanism of action Coagulant Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Uitsluitend in geaccrediteerde hemofiliebehandelcentra. Additional remarks Recombinant Von Willebrand factor. Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP No Submission date 2025 Expected Registration January 2026 Orphan drug No Registration phase Registered Reimbursement Reimbursed from the basic package Medicine sluice Rejected Additional remarks Positieve CHMP-opinie in november 2025. Therapeutic value Current treatment options Meerdere uit bloedplasma bereidde von Willebrand factor concentraten: bijvoorbeeld: Haemate-P, Wilate, Wilfactin. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er zijn nog geen resultaten bekend. Duration of treatment Frequency of administration Dosage per administration References NCT05582993 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie Additional remarks De werkgroep verwacht dat er niet meer dan 50 patiënten in aanmerking komen. Expected cost per patient per year Cost € 11,000.00 - 26,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GIPdatabank Additional remarks Op basis van declaratiedata van bestaande von Willebrand factoren zullen de kosten tussen de €11.000 en €26.000 per patiënt per jaar zitten. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks