Extended indication Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic c
Therapeutic value Possibly no place in the treatment regimen
Total cost 852,440.00
Registration phase Registered and not reimbursed

Product

Active substance Pemigatinib
Domain Oncology
Reason of inclusion New medicine (specialité)
Main indication Liver cancer
Extended indication Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
Proprietary name Pemazyre
Manufacturer Incyte
Portfolio holder Incyte
Mechanism of action Tyrosine kinase inhibitor
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional comments FGFR 2 fusion positivity status must be known prior to initiation of Pemazyre therapy. Assessment for FGFR 2 fusion positivity in tumor specimen should be performed with an appropriate diagnostic test.

Registration

Registration route Centralised (EMA)
Type of trajectory Conditional marketing authorisation
ATMP No
Submission date November 2019
Expected Registration March 2021
Orphan drug Yes
Registration phase Registered and not reimbursed
Additional comments Positieve CHMP-opinie in januari 2021.

Therapeutic value

Current treatment options Gemcitabine en cisplatine in de eerstelijn, geen geregistreerde geneesmiddelen voor de tweedelijn.
Therapeutic value Possibly no place in the treatment regimen
Substantiation Voldoet niet aan cieBOM NRS criteria;
Duration of treatment Median 8.1 month / months
Frequency of administration 1 times a day
Dosage per administration 13.5 mg
References SmPC, FIGHT-202 (NCT02924376); --https://www.fiercepharma.com/marketing/incyte-scores-fda-nod-for-rare-bile-duct-cancer-therapy-pemazyre. -https://www.thelancet.com/pdfs/journals/lanonc/PIIS1470-2045(20)30109-1.pdf
Additional comments Once daily for 14 days followed by 7 days off therapy

Expected patient volume per year

Patient volume

10

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 80,000.00 - 90,488.00
References Fabrikant: Data on file. ZN-aanvraagformulier NZa aanvraagformulier
Additional comments Lijstprijs €7.732

Potential total cost per year

Total cost

852,440.00

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 primary completion date october 2023

Other information

There is currently no futher information available.