Fedratinib Medicine 12 June 2018 Extended indication Myelofibrosis Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Fedratinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Myeloproliferative disorders Extended indication Myelofibrosis Current proprietary name Already available biosimilars / generics Proprietary name Inrebic Manufacturer Celgene Portfolio holder Mechanism of action JAK tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks JAK2 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date February 2020 Expected Registration March 2021 Orphan drug Unknown Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks In 2013 zijn de klinische studies door Sanofi stopgezet wegens ernstige bijwerkingen (Wernicke’s encephalopathie). Celgene heeft federatinib inmiddels opgekocht. Therapeutic value Current treatment options Ruxolitinib Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Milt verkleining, symptom score reduction Duration of treatment Frequency of administration 1 times a day Dosage per administration 400 mg References JAKARTA: NCT01437787 JAKARTA2: NCT01523171 Additional remarks Expected patient volume per year Patient volume 20 - 143 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks 143 diagnoses in 2016. De inschatting van experts is 20 patiënten. Expected cost per patient per year Cost References medicijnkosten.nl en gipdatabank.nl Additional remarks De kosten zullen mogelijk in dezelfde range komen te liggen als ruxolitinib, wat per tablet van 10 mg €64,80 kost (inclusief BTW). Uiteindelijke kosten per patiënt per jaar afhankelijk van de dosis en behandelduur. De kosten van ruxolitinib betroffen gemiddeld genomen bij de behandeling van myelofibrose zo'n €30.000 per patiënt per jaar. 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 Polycythemia Vera References adisinsight Additional remarks Geen lopende fase 3 studies. Other information Additional remarks