Extended indication Treatment of locally advanced or metastatic cholangiocarcinoma relapsed or refractory after at least
Therapeutic value No judgement
Total cost 1,280,000.00
Registration phase Registration application pending


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. Alleen de patiënten met een FGFR2 mutatie komen in aanmerking.
Proprietary name Pemazyre
Manufacturer Incyte
Mechanism of action Tyrosine kinase inhibitor
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 de eerstelijn, geen geregistreerde geneesmiddelen voor de tweedelijn.
Therapeutic value No judgement
Duration of treatment Median 7.2 month / months
Frequency of administration 1 times a day
Dosage per administration 13,5mg
References FIGHT-202 (NCT02924376); https://www.fiercepharma.com/marketing/incyte-scores-fda-nod-for-rare-bile-duct-cancer-therapy-pemazyre.

Expected patient volume per year

Patient volume


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 Er zijn ongeveer 700 patiënten in Nederland gediagnosticeerd met een cholangiocarcinoom. Hiervan betreft het in 36% een intrahepatisch carcinoom (n=252), waarvan 237 patiënten unresectable/relapsed resectable zijn. Hiervan zal ongeveer 50% chemotherapie ontvangen (n-119). 50% van de patiënten zal getest worden op een FGFR2 mutatie, waarvan ongeveer 10%-16% positief is, dit betekent dat er rond de 10 patiënten in aanmerking komen voor deze behandeling.

Expected cost per patient per year

Cost 120,000.00 - 136,000.00
References https://www.fiercepharma.com/marketing/incyte-scores-fda-nod-for-rare-bile-duct-cancer-therapy-pemazyre.
Additional comments In de Verenigde Staten worden de kosten per patiënt per jaar ingeschat op $136.000-153.000, dit is ongeveer €120.000-€136.000.

Potential total cost per year

Total cost


This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Eerste lijns behandeling bij dezelfde indicatie.
References FIGHT-302 trial; Clinicaltrials.gov NCT03656536
Additional comments Estimated study completion Q4 2024.

Other information

There is currently no futher information available.