Extended indication Tovorafenib monotherapy for second line or later treatment of relapsed or refractory BRAF-altered lo
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Tovorafenib
Domain Oncology
Reason of inclusion New medicine (specialité)
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.
Proprietary name Ojemda
Manufacturer Day One
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Fabrikant: Day One. Kinaseremmer.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Therapeutic value No estimate possible yet
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

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
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

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.