Efanesoctocog alfa Medicine 06 June 2023 Extended indication Treatment and prophylaxis of bleeding in patients with haemophilia A Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Efanesoctocog alfa Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Treatment and prophylaxis of bleeding in patients with haemophilia A Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sobi Portfolio holder Sobi Mechanism of action Coagulant Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Factor VIII replacement. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date May 2023 Expected Registration June 2024 Orphan drug Yes Registration phase Registration application pending 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 Duration of treatment Frequency of administration 1 times a week Dosage per administration 50 UI/kg References NCT05817812 (Freedom) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost € < 260,000.00 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 https://www.biopharmadive.com/news/sanofi-bioverativ-fda-approve-drug-rare-anemia/618416/ Additional remarks In de Verenigde Staten is de lijstprijs bekend. Dit is 280.000 dollar per patiënt per jaar voor een gemiddelde patiënt van 75 kg (1800 dollar per dosis). Omgerekend naar euro is dat €260.000 . De prijs in Nederland is nog niet bekend. Waarschijnlijk zal deze prijs lager zijn in Nederland. 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