Active substance
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.

1. Product

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

2. Registration

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

3. 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
FIGHT-202 (NCT02924376)

4. Expected patient volume per year

Patient volume

< 156

Market share is generally not included unless otherwise stated.

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%).

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

Indication extension
Additional comments
Enkel meerdere lopende fase 2 studies.

9. Other information

There is currently no futher information available.