Selumetinib Medicine 03 June 2025 Extended indication Selumetinib monotherapy for first line treatment of symptomatic inoperable plexiform neurofibroma in adults and elderly with neurofibromatosis type 1. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Selumetinib Domain Oncology Reason of inclusion Indication extension IND Main indication Oncology other Extended indication Selumetinib monotherapy for first line treatment of symptomatic inoperable plexiform neurofibroma in adults and elderly with neurofibromatosis type 1. Current proprietary name Already available biosimilars / generics Proprietary name Koselugo Manufacturer Alexion Portfolio holder Alexion Mechanism of action MEK kinase inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Erasmus MC Additional remarks 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 Frequency of administration 2 times a day Dosage per administration 25mg/m2 BSA References NCT04924608 (KOMET) 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 € < 267,402.00 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 Advies - vergoed selumetinib (Koselugo®) voor de behandeling van goedaardige tumoren bij kinderen met neurofibromatose type 1 (NF1). Zorginstituut Nederland. 2024 (1). Additional remarks Een behandeling voor een patiënt met een gemiddeld lichaamsoppervlak van 1,70m2 kost €267.402 per patiënt per jaar (1). 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