Pemigatinib Medicine 06 June 2023 Extended indication Extension of indication to include treatment of adults with myeloid/lymphoid neoplasms (MLNs) with Fibroblast Growth Factor Receptor1 (FGFR1) rearrangement. Therapeutic value No estimate possible yet Total cost € 12,150,000.00 Registration phase Registration application pending Product Active substance Pemigatinib Domain Hematology Reason of inclusion Indication extension IND 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. Current proprietary name Already available biosimilars / generics 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) Centre of expertise 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 Particularity Unknown ATMP No Submission date January 2024 Expected Registration November 2024 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options 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 This assessment does not indicate any potential inclusion in the package. Substantiation De laatste resultaten van de FIGHT-203 studie (2022 ASH) lieten in patiënten met een FGFR-1 mutatie in patiënten, 28 (73,7%) een complete klinische respons zien (CR; primary endpoint). De mate van cytogenetische respons (CCyR) was 70,0% (n=28/40). De CRs en CCyRs in eerder behandelde patiënten was, 75,8% (n=25/33) en 71.4% (n=25/35), De mediane behandeltijd met pemigatinib was 6,3 maanden (range: 0.5-50.2). Duration of treatment Median 6.3 month / months Frequency of administration 1 times a day Dosage per administration 13,5 mg References 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. 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 < 180 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks MLN met FGFR1 mutatie is zeldzaam. De incidentie is ongeveer <1 op de 100.000 personen per jaar. Omgerekend naar Nederland zou dit betekenen dat er hooguit 180 mensen in aanmerking zullen 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 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. References Additional remarks Lijstprijs €7.732 Potential total cost per year Total cost € 12,150,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks