Eptacog alfa (activated) Medicine 13 December 2018 Extended indication Extension of Indication to extend patient population of NovoSeven for use in patients with Glanzmann’s thrombasthenia with or present refractoriness to platelet transfusions, or where platelets are not readily available. Therapeutic value Possible added value Total cost Registration phase Registered and reimbursed Product Active substance Eptacog alfa (activated) Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Hemostasis promoting medication Extended indication Extension of Indication to extend patient population of NovoSeven for use in patients with Glanzmann’s thrombasthenia with or present refractoriness to platelet transfusions, or where platelets are not readily available. Current proprietary name Already available biosimilars / generics Proprietary name NovoSeven Manufacturer Novo Nordisk Portfolio holder Mechanism of action Coagulant Route of administration Intravenous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Factor VIIa stimulants; Factor X stimulants Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date May 2018 Expected Registration December 2018 Orphan drug Unknown Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie november 2018. Geregistreerd in december 2018. Therapeutic value Current treatment options Huidige behandeling bestaat uit trombocytentransfusie. Bij patiënten met antistoffen tegen GPIIb/IIIa is trombocytentransfusie niet mogelijk, maar bij patiënten zonder antistoffen (huidige uitbreiding van de indicatie) in principe wel. Als er geen beschikking is over trombocytentransfusie is er geen goede alternatieve behandeling. Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Bij behandling met trombocytentransfusie bij Glanzmann is er grote kans op ontwikkelen van antistoffen tegen GPIIb/IIIa. Dit probleem is er niet met eptacog alfa. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume 10 - 20 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://pfhaemophilianl.pfizersite.io/ziekte-glanzmann-altijd-beperkt-niet Additional remarks Er zijn ongeveer 30-40 patiënten met de ziekte van Glanzmann in Nederland. Onduidelijk welk aantal patiënten onder de uitbreiding van indicatie valt. Inschatting: 10-20 patiënten. Expected cost per patient per year Cost References medicijnkosten.nl Additional remarks kosten voor 1 injectieflacon van met poeder voor inectievloeistof 1mg FL, bedraagt € 797,45 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