Pemigatinib Medicine 10 June 2020 Extended indication Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy. Therapeutic value No estimate possible yet Total cost € 1,280,000.00 Registration phase Registered Product Active substance Pemigatinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Liver cancer Extended indication Pemazyre monotherapy is indicated for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy. Current proprietary name Already available biosimilars / generics Proprietary name Pemazyre Manufacturer Incyte Portfolio holder Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fibroblast growth factor receptor 2 (FGFR2). Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date January 2020 Expected Registration March 2021 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in januari 2021. Therapeutic value Current treatment options Gemcitabine en cisplatine in de eerstelijn, geen geregistreerde geneesmiddelen voor de tweedelijn. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Median 7.2 month / months Frequency of administration 1 times a day Dosage per administration 13,5mg References FIGHT-202 (NCT02924376); https://www.fiercepharma.com/marketing/incyte-scores-fda-nod-for-rare-bile-duct-cancer-therapy-pemazyre. Additional remarks Expected patient volume per year Patient volume 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Plentz RR. Systemic Therapy of Cholangiocarcinoma. Visc Med. 2016;32(6):427–430. Additional remarks Er zijn ongeveer 700 patiënten in Nederland gediagnosticeerd met een cholangiocarcinoom. Hiervan betreft het in 36% een intrahepatisch carcinoom (n=252), waarvan 237 patiënten unresectable/relapsed resectable zijn. Hiervan zal ongeveer 50% chemotherapie ontvangen (n-119). 50% van de patiënten zal getest worden op een FGFR2 mutatie, waarvan ongeveer 10%-16% positief is, dit betekent dat er rond de 10 patiënten in aanmerking komen voor deze behandeling. Expected cost per patient per year Cost € 120,000.00 - 136,000.00 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 https://www.fiercepharma.com/marketing/incyte-scores-fda-nod-for-rare-bile-duct-cancer-therapy-pemazyre. Additional remarks In de Verenigde Staten worden de kosten per patiënt per jaar ingeschat op $136.000-153.000, dit is ongeveer €120.000-€136.000. Potential total cost per year Total cost € 1,280,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Eerste lijns behandeling bij dezelfde indicatie. References FIGHT-302 trial; Clinicaltrials.gov NCT03656536 Additional remarks Estimated study completion quarter of 2024. Other information Additional remarks