Pemigatinib

Extended indication	Treatment of locally advanced or metastatic cholangiocarcinoma relapsed or refractory after at least one line of systemic therapy.
Therapeutic value	No judgement
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.
Manufacturer	Incyte
Mechanism of action	Receptor antagonist
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Intermural (MSZ)
Additional remarks	Fibroblast growth factor receptor 2 (FGFR2)

Registration

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
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 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%).

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 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

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 remarks	Enkel meerdere lopende fase 2 studies.

Other information

There is currently no futher information available.