Selumetinib Medicine 04 December 2025 Extended indication Koselugo as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1) aged 1 year to less than 7 years and for older patients with swallowing difficulties. Therapeutic value Total cost Registration phase Positive CHMP opinion Product Active substance Selumetinib Domain Oncology Reason of inclusion Indication extension IND Main indication Extended indication Koselugo as monotherapy is indicated for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in patients with neurofibromatosis type 1 (NF1) aged 1 year to less than 7 years and for older patients with swallowing difficulties. 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 Granulate Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date January 2025 Expected Registration January 2026 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Broadly excluded Additional remarks Deze indicatieuitbreiding geldt alleen voor de nieuwe toedieningsvorm (granulaat in capsules voor orale toediening). Selumetinib is in november 2025 in de sluis geplaatst, voor zover verstrekt in het kader van alle geneeskundige behandelingen, met uitzondering van de toepassing tot 1 januari 2028 als monotherapie in het kader van de geneeskundige behandeling van symptomatische, inoperabele plexiforme neurofibromen bij pediatrische patiënten met neurofibromatose type 1 in de leeftijd van drie jaar of ouder (harde capsules). Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration 5 of 7,5 mg References NCT05309668 (SPRINKLE) 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 References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks