Pemigatinib Geneesmiddel 06 June 2023 Uitgebreide indicatie Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with Fibroblast Growth Factor Receptor1 (FGFR1) rearrangement. 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 IND 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. Huidig specialité Reeds beschikbare biosimilars/generieken Merknaam Pemazyre Fabrikant Incyte Portfoliohouder Incyte Werkingsmechanisme Tyrosine kinase remmer Toedieningsweg Oraal Toedieningsvorm Tablet Bekostigingskader Intramuraal (MSZ) Expertisecentrum 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 Bijzonderheid Onbekend ATMP Nee Indieningsdatum December 2023 Verwachte registratie Februari 2025 Weesgeneesmiddel Ja Registratiefase Registratieaanvraag in behandeling Vergoeding Geneesmiddelensluis 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 This assessment does not indicate any potential inclusion in the package. 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 Market share is generally not included unless otherwise stated. Maximaal patiëntvolume voor sluis 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 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. Bronnen 65.000 tot 70.000 maximaal per jaar Aanvullende opmerkingen Lijstprijs €7.732 Mogelijke totale kosten per jaar Totale kosten € 67,500.00 Totale kosten voor sluis Aanvullende opmerkingen Off-label gebruik Indicatieuitbreidingen Onbekend Indicaties off-label gebruik Bronnen Aanvullende opmerkingen Indicatieuitbreiding Indicatieuitbreidingen Onbekend Indicatieuitbreidingen Bronnen Aanvullende opmerkingen Overige informatie Aanvullende opmerkingen