Extended indication

Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with F

Therapeutic value

No estimate possible yet

Total cost


Registration phase

Registration application pending


Active substance




Reason of inclusion

Indication extension

Main indication

Myeloproliferative disorders

Extended indication

Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with Fibroblast Growth Factor Receptor1 (FGFR1) rearrangement.

Proprietary name




Portfolio holder


Mechanism of action

Tyrosine kinase inhibitor

Route of administration


Therapeutical formulation


Budgetting framework

Intermural (MSZ)

Additional remarks
FGFR-1 rearrangement positivity status must be known prior to initiation of Pemazyre therapy. Assessment for FGFR 1 rearrangement positivity in tumor specimen should be performed with an appropriate diagnostic test.


Registration route

Centralised (EMA)

Type of trajectory

Normal trajectory



Submission date

January 2024

Expected Registration

November 2024

Orphan drug


Registration phase

Registration application pending

Therapeutic value

Current treatment options

For patients with MLN with FGFR1 rearrangement in chronic phase, the National Comprehensive Cancer Network (NCCN) US-guidelines identify enrolment in clinical trials as the preferred treatment option. In the absence of a clinical trial, the current recommended approach is monotherapy with a tyrosine kinase inhibitor (TKI) with activity against FGFR1.The NCCN guidelines list pemigatinib, midostaurin, and ponatinib as TKIs with activity against FGFR1. However, none of these drugs (except pemigatinib in US) have been approved for the treatment of MLN with FGFR1 rearrangement.

Therapeutic value

No estimate possible yet


De laatste resultaten van de FIGHT-203 studie (2022 ASH) lieten in patiënten met een FGFR-1 mutatie in patiënten, 28 (73,7%) een complete klinische respons zien (CR; primary endpoint). De mate van cytogenetische respons (CCyR) was 70,0% (n=28/40). De CRs en CCyRs in eerder behandelde patiënten was, 75,8% (n=25/33) en 71.4% (n=25/35), De mediane behandeltijd met pemigatinib was 6,3 maanden (range: 0.5-50.2).

Duration of treatment

Median 6.3 month / months

Frequency of administration

1 times a day

Dosage per administration

13,5 mg

ASH 2022 presentation details: Lead Author: Srdan Verstovsek, poster P1732: FIGHT-203, an ongoing phase 2 study of pemigatinib in patients with myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) rearrangement (MLNFGFR1): a focus on centrally reviewed clinical and cytogenetic responses in previously treated patients. ClinicalTrials.gov. FIGHT-203. Accessed Dec 2022. https://clinicaltrials.gov/ct2/show/NCT03011372.
Additional remarks
Latest results of the FIGHT-203 study (presented at 2022 ASH) showed in treatment naïve or previously treated patients with MLN with FGFR-1 rearrangements: Of 38 evaluable patients, 28 (73.7%) achieved a clinical complete response (CR; primary endpoint). The rate of complete cytogenetic responses (CCyR) was 70.0% (n=28/40). The rates of CRs and CCyRs in previously treated, efficacy-evaluable patients were 75.8% (n=25/33) and 71.4% (n=25/35), respectively. Of 21 patients with chronic phase (CP) disease, 18 (85.7%) exhibited both clinical CRs and CCyRs. Of 17 patients with blast phase (BP) disease, 9 presented with clinical CRs (52.9%), and 8 (47.1%) with CCyRs. The median exposure to pemigatinib treatment was 6.3 months (range: 0.5-50.2).

Expected patient volume per year

Patient volume

< 180

Market share is generally not included unless otherwise stated.

Additional remarks
MLN met FGFR1 mutatie is zeldzaam. De incidentie is ongeveer <1 op de 100.000 personen per jaar. Omgerekend naar Nederland zou dit betekenen dat er hooguit 180 mensen in aanmerking zullen komen. Er moet eerst een diagnostische test afgenomen worden om vast te stellen dat de patiënt de mutatie heeft.

Expected cost per patient per year


65,000.00 - 70,000.00

Additional remarks
Lijstprijs €7.732

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.