Pemigatinib Geneesmiddel 06 June 2023 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 IND Hoofdindicatie Myeloproliferatieve aandoeningen Uitgebreide indicatie Myeloid/Lymphoid Neoplasms ((8p11 var) 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 November 2023 Verwachte registratie Juli 2024 Weesgeneesmiddel Ja Registratiefase Klinische studies 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 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 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 Market share is generally not included unless otherwise stated. Maximaal patiëntvolume voor sluis Bronnen 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 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 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