Tovorafenib Medicine 03 December 2024 Extended indication Tovorafenib monotherapy for second line or later treatment of relapsed or refractory BRAF-altered low-grade glioma in infants and toddlers from 6 months, children, adolescents and adults up to 25 years of age. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Tovorafenib Domain Oncology Reason of inclusion New medicine (specialité) Main indication Unknown Extended indication Tovorafenib monotherapy for second line or later treatment of relapsed or refractory BRAF-altered low-grade glioma in infants and toddlers from 6 months, children, adolescents and adults up to 25 years of age. Current proprietary name Already available biosimilars / generics Proprietary name Ojemda Manufacturer Day One Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fabrikant: Day One. Kinaseremmer. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date 2025 Expected Registration 2026 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 Duration of treatment Median 15.8 month / months Frequency of administration 1 times a week Dosage per administration 380 mg/m2 (max. 600 mg) References NCT04775485 (FIREFLY-1) (1); Kilburn et al. Nat Med. 2024 (2). Additional remarks The recommended dosage of OJEMDA based on body surface area (BSA) is 380mg/m2 orally once weekly (the maximum recommended dosage is 600mg orally once weekly) with or without food. Median duration of treatment was 15,8 months (2). Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Prinses Máxima Centrum (1). Additional remarks In Nederland krijgen elk jaar ongeveer 40 kinderen een laaggradig glioom (1). Expected cost per patient per year Cost € < 640,309.00 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 Drugs.com (1) Additional remarks In de Verenigde Staten kosten 16 tabletten van 100mg $35,723 (1). Omgerekend is dit ongeveer €32.672, maar dit getal kan lager uitvallen omdat medicijnen doorgaans duurder zijn in de Verenigde Staten. Uitgaande van een mediane behandelduur van 15,8 maanden en een wekelijkse toediening van 600mg kan een schatting gemaakt worden van €640.309 per patiënt per jaar (€32.672/16∗6∗52,18). Hierbij is nog geen rekening gehouden met een eventueel stapeleffect in verband met de behandelduur die langer dan een jaar duurt. 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 Unknown Indication extensions References Additional remarks Other information Additional remarks