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Pemigatinib

Pemazyre

Uitgebreide indicatie Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with F
Therapeutische waarde Nog geen inschatting mogelijk
Totale kosten 67.500,00
Registratiefase Registratieaanvraag in behandeling

Product

Werkzame stof Pemigatinib
Domein Hematologie
Reden van opname Indicatieuitbreiding
Hoofdindicatie Myeloproliferatieve aandoeningen
Uitgebreide indicatie Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with Fibroblast Growth Factor Receptor1 (FGFR1) rearrangement.
Merknaam Pemazyre
Fabrikant Incyte
Portfoliohouder Incyte
Werkingsmechanisme Tyrosine kinase remmer
Toedieningsweg Oraal
Toedieningsvorm Tablet
Bekostigingskader Intramuraal (MSZ)
Aanvullende opmerkingen 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.

Registratie

Registratieroute Centraal (EMA)
Type traject Normaal traject
ATMP Nee
Indieningsdatum December 2023
Verwachte registratie Februari 2025
Weesgeneesmiddel Ja
Registratiefase Registratieaanvraag in behandeling
Aanvullende opmerkingen 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.

Therapeutische waarde

Huidige behandelopties 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.
Therapeutische waarde

Nog geen inschatting mogelijk

Deze inschatting doet geen uitspraak over de mogelijke opname in het pakket.
Onderbouwing 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.
Behandelduur Mediaan 9 maand/maanden
Toedieningsfrequentie 1 maal per dag
Dosis per toediening 13,5 mg
Bronnen 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.
Aanvullende opmerkingen 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).

Verwacht patiëntvolume per jaar

Patiëntvolume

< 1

Marktaandeel wordt in de regel niet meegenomen tenzij anders vermeld.
Bronnen 1. Colucci, 2022
Aanvullende opmerkingen 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.

Verwachte kosten per patiënt per jaar

Kosten 65.000,00 - 70.000,00
Bronnen 65.000 tot 70.000 maximaal per jaar
Aanvullende opmerkingen Lijstprijs €7.732

Mogelijke totale kosten per jaar

Totale kosten

67.500,00

Dit bedrag geeft een indicatie van de totale kosten. Het is de uitkomst van het gemiddelde verwacht patiëntvolume maal de gemiddelde kosten per patiënt; beiden per jaar.

Off-label gebruik

Er is op dit moment niets bekend over off-label gebruik.

Indicatieuitbreiding

Er is op dit moment niets bekend over indicatieuitbreidingen.

Overige informatie

Er is op dit moment geen overige informatie.