Pemigatinib Medicine 10 June 2020 Extended indication Treatment of locally advanced or metastatic cholangiocarcinoma relapsed or refractory after at least one line of systemic therapy. Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Pemigatinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Liver cancer Extended indication Treatment of locally advanced or metastatic cholangiocarcinoma relapsed or refractory after at least one line of systemic therapy. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Incyte Portfolio holder Mechanism of action Receptor antagonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fibroblast growth factor receptor 2 (FGFR2) Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date January 2020 Expected Registration February 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Gemcitabine en cisplatine in eerstelijn Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References FIGHT-202 (NCT02924376) Additional remarks Expected patient volume per year Patient volume < 156 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Plentz RR. Systemic Therapy of Cholangiocarcinoma. Visc Med. 2016;32(6):427–430. Additional remarks Volgens het NKR werd er in 2017 156 keer een cholangiocarcinoom in Nederland gediagnosticeerd. Dit is het maximale aantal patiënten dat in aanmerking kan komen voor pemigatinib. Mogelijk dat alleen de patiënten met een FGFR2 mutatie in aanmerking komen (ongeveer zo'n 16%). 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 No Indication extensions References Adisinsight Additional remarks Enkel meerdere lopende fase 2 studies. Other information Additional remarks