Eptacog beta (activated) Medicine 12 June 2018 Extended indication CEVENFACTA is indicated in adults and adolescents (12 years of age and older) for the treatment of bleeding episodes and for the prevention of bleeding in those undergoing surgery or invasive procedures in the following patient groups: - in patients with congenital haemophilia with high-responding inhibitors to coagulation factors VIII or IX (i.e. ≥5 Bethesda Units (BU)); - in patients with congenital haemophilia with low titre inhibitors (BU <5), but expected to have a high anamnestic response to factor VIII or factor IX administration or expected to be refractory to increased dosing of FVIII or FIX. Therapeutic value Possible equal value Total cost € 342,000.00 Registration phase Positive CHMP opinion Product Active substance Eptacog beta (activated) Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Hemostasis promoting medication Extended indication CEVENFACTA is indicated in adults and adolescents (12 years of age and older) for the treatment of bleeding episodes and for the prevention of bleeding in those undergoing surgery or invasive procedures in the following patient groups: - in patients with congenital haemophilia with high-responding inhibitors to coagulation factors VIII or IX (i.e. ≥5 Bethesda Units (BU)); - in patients with congenital haemophilia with low titre inhibitors (BU <5), but expected to have a high anamnestic response to factor VIII or factor IX administration or expected to be refractory to increased dosing of FVIII or FIX. Current proprietary name Already available biosimilars / generics Proprietary name Cevenfacta Manufacturer rEVO Portfolio holder Mechanism of action Coagulant Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Meer informatie over de werking van het product: "A transgenically produced recombinant form of human factor VIIa." Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date February 2021 Expected Registration July 2022 Orphan drug No Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen in mei 2022. Therapeutic value Current treatment options Eptacog alfa, emicizumab, protrombinecomplexen Therapeutic value Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation Het verschil tussen eptacog alfa in tegenstelling tot eptacog beta is dat het geproduceerd is op transgene wijze. Daardoor zou het een stabielere / natuurlijkere glycosylering hebben dan het recombinant geproduceerde eptacog alfa. Echter klinische vergelijking tussen deze twee ontbreekt. Betreft dan dus grotendeels substitutie. Duration of treatment Frequency of administration Dosage per administration References NCT02548143 Additional remarks PERSEPT 1: "Intravenous 75 μg/kg or 225 μg/kg initial doses with 75 μg/kg subsequent doses by schedule were administered until clinical response." Expected patient volume per year Patient volume < 30 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NVHB Additional remarks Aantallen patiënten hemofilie A met remmers wordt door de behandelaren (NVHB) ingeschat op maximaal totaal 30 patiënten. Expected cost per patient per year Cost € 5,700.00 - 17,100.00 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 Medicijnkosten.nl Additional remarks De verwachting is dat de prijs vergelijkbaar zal zijn met eptacog alfa (€820 voor een spuit van 1mg). Per toediening zal er gemiddeld 7mg moeten worden toegediend (€5.700). Bij bloedingen 1-3 toedieningen (maximaal €17.100), bij profylaxe meermaals. Potential total cost per year Total cost € 342,000.00 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