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Pemigatinib

Pemazyre

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 67,500.00
Registration phase Registration application pending

Product

Active substance Pemigatinib
Domain Hematology
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 Pemazyre
Manufacturer Incyte
Portfolio holder Incyte
Mechanism of action Tyrosine kinase inhibitor
Route of administration Oral
Therapeutical formulation Tablet
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

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date December 2023
Expected Registration February 2025
Orphan drug Yes
Registration phase Registration application pending
Additional remarks Registration will be based on results of the FIGHT-203 study: a single arm, open-label phase 2 study evaluating the efficacy and safety of pemigatinib monotherapy in patients with MLN with FGFR1 rearrangement.

Therapeutic value

Current treatment options There are currently no EU clinical guidelines for the management of MLNs with FGFR1 rearrangement. 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
Substantiation Updated results of the FIGHT-203 study (presented at 2024 EHA) showed in treatment naïve or previously treated patients with MLN with FGFR-1 rearrangements: Of 45 evaluable patients, 31 (68.9%) achieved a clinical complete response (CR; primary endpoint). The rate of complete cytogenetic responses (CCyR) was 71.1% (n=32/45). As assessed by investigator. 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 24 patients with chronic phase (CP) disease, 22 (91.7%) presented with clinical CRs and 20 (83.3%) with CCyRs. Of 18 patients with blast phase (BP) disease, 6 (33.3%) presented with clinical CRs, and 9 (50.0%) with CCyRs. The median exposure to pemigatinib treatment was 6.3 months (range: 0.5-50.2). Latest results FIGHT-203 (Vannuchi A. et al. EHA 2024 Poster 1042): CR rate: 73.8% (n=42); CCyR rate 71.1%; as assessed by CRC.
Duration of treatment Median 9 month / months
Frequency of administration 1 times a day
Dosage per administration 13,5 mg
References EHA 2024 presentation details: Lead Author: Vannuchi AM et al, poster P1042: FIGHT-203, a phase 2 study of pemigatinib in patients with myeloid/lymphoid neoplasms (MLNs) with fibroblast growth factor receptor 1 (FGFR1) gene rearrangement. ClinicalTrials.gov. FIGHT-203. Accessed June 2024. 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

< 1

Market share is generally not included unless otherwise stated.
References 1. Colucci, 2022
Additional remarks MLNs with FGFR1 rearrangement are an aggressive, ultra-rare cancer estimated to affect around 0.018 people per 100,000 population (i.e. 1 per 5m) and are associated with a median overall survival of <1 year. (1) Omgerekend naar Nederland zou dit betekenen dat er hooguit 1 patiënt per jaar in aanmerking zal 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

Cost 65,000.00 - 70,000.00
References 65.000 tot 70.000 maximaal per jaar
Additional remarks Lijstprijs €7.732

Potential total cost per year

Total cost

67,500.00

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.