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Pemigatinib

Pemazyre

Uitgebreide indicatie Myeloid/Lymphoid Neoplasms ((8p11 var)
Therapeutische waarde Nog geen inschatting mogelijk
Totale kosten 67.500,00
Registratiefase Klinische studies

Product

Werkzame stof Pemigatinib
Domein Hematologie
Reden van opname Indicatieuitbreiding
Hoofdindicatie Myeloproliferatieve aandoeningen
Uitgebreide indicatie Myeloid/Lymphoid Neoplasms ((8p11 var)
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 November 2023
Verwachte registratie Juli 2024
Weesgeneesmiddel Ja
Registratiefase Klinische studies
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 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 Not assessed, no approved treatment for MLN with FGFR-1 rearrangement available. 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).
Behandelduur Mediaan 6.3 maand/maanden
Toedieningsfrequentie 1 maal per dag
Dosis per toediening 13,5 mg
Bronnen 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.
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.
Aanvullende opmerkingen Very rare disease. No published estimates of incidence or prevalence Incidence estimated: <1 case per 100.000 persons/year.

Verwachte kosten per patiënt per jaar

Kosten 65.000,00 - 70.000,00
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.