Extended indication

Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic ch

Therapeutic value

No estimate possible yet

Total cost

1,280,000.00

Registration phase

Registered

Product

Active substance

Pemigatinib

Domain

Oncology and Hematology

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 is relapsed or refractory after at least one line of systemic therapy.

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

Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory

Submission date

January 2020

Expected Registration

March 2021

Orphan drug

Yes

Registration phase

Registered

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

No estimate possible yet

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

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

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

1,280,000.00

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 study completion quarter of 2024.

Other information

There is currently no futher information available.