Selumetinib Medicine 12 June 2018 Extended indication Paedriatric (12-18 years of age) neurofibromatosis type-1. Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Selumetinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Neuroendocrine cancer Extended indication Paedriatric (12-18 years of age) neurofibromatosis type-1. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer AstraZeneca Portfolio holder Mechanism of action MEK kinase inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Anti MAPK kinase (MEK). Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date June 2020 Expected Registration June 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Fabrikant verwacht een indiening in H1 2020 en registratie in H1 2021. Therapeutic value Current treatment options Geen effectieve behandeling beschikbaar. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 30 month / months Frequency of administration 2 times a day Dosage per administration 25 mg/m2 References NCT01362803; Dombi A. et al. N. Engl. J. Med. 2016 375:2550-60 Additional remarks Expected patient volume per year Patient volume < 500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NHG; CBS Additional remarks De incidentie van NF1 bij het aantal levendgeborenen is 1 op de 2.500. Als dit doorberekend wordt naar de populatie van 12 tot 18 jaar dan komt dit neer op een kleine 500 patiënten. Hiervan komt alleen het deel in aanmerking dat niet voldoende behandeld kan worden met een chirurgische ingreep. 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 Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Mogelijke indicatie-uitbreiding: Differentiated thyroid cancer - first-line with radioactive iodine References SPS Additional remarks Other information Additional remarks