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 € 67,500.00 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 December 2023 Expected Registration February 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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. Therapeutic value Current treatment options 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. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation 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. Duration of treatment Median 9 month / months Frequency of administration 1 times a day Dosage per administration 13,5 mg References 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. 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 < 1 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1. Colucci, 2022 Additional remarks 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. 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 65.000 tot 70.000 maximaal per jaar Additional remarks Lijstprijs €7.732 Potential total cost per year Total cost € 67,500.00 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