Active substance Pemigatinib
Domain Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen 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.


Manufacturer Incyte
Mechanism of action Receptor antagonist
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional comments Fibroblast growth factor receptor 2 (FGFR2)


Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date January 2020
Expected Registration February 2021
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Current treatment options Gemcitabine en cisplatine in eerstelijn
Therapeutic value No judgement
Duration of treatment Not found
Frequency of administration 1 times a day
References FIGHT-202 (NCT02924376)

Expected patient volume per year

Patient volume

< 156

Market share is generally not included unless otherwise stated.

References NKR; Plentz RR. Systemic Therapy of Cholangiocarcinoma. Visc Med. 2016;32(6):427–430.
Additional comments 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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References Adisinsight
Additional comments Enkel meerdere lopende fase 2 studies.

Other information

There is currently no futher information available.