Ziftomenib Medicine 03 June 2025 Extended indication Ziftomenib monotherapy for second line or later treatment of relapsed/refractory NPM1-mutant acute myeloid leukaemia in adults and elderly who have failed or are ineligible for any approved standard of care therapies, including HSCT. Therapeutic value No estimate possible yet Total cost € 21,760,000.00 Registration phase Clinical trials Product Active substance Ziftomenib Domain Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Ziftomenib monotherapy for second line or later treatment of relapsed/refractory NPM1-mutant acute myeloid leukaemia in adults and elderly who have failed or are ineligible for any approved standard of care therapies, including HSCT. Current proprietary name Already available biosimilars / generics Proprietary name Komzifti Manufacturer Kura Oncology Portfolio holder Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: M=menin-mixed lineage leukemia fusion protein inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Submission date June 2026 Expected Registration July 2027 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Op dit moment kan er nog niet veel gezegd worden over de data uit de fase 3 studie. Ook revumenib is een menin inhibitor die voor dezelfde indicatie in de nabije toekomst op de markt verwacht wordt. Er wordt door experts veel van dit nieuwe werkingsmechanisme verwacht. Duration of treatment Frequency of administration Dosage per administration References NCT04067336 (KO-MEN-001) Additional remarks Expected patient volume per year Patient volume 256 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR 2021 (1); NPM 1 Mutations in AML—The Landscape in 2023. Naman Sharma, Jane L Liesveld. (2) Additional remarks Er waren 853 AML diagnoses in 2021 (1). NPM1 mutaties worden gezien bij ongeveer 30% van gediagnosticeerde AML gevallen (2). Er zouden dan in theorie 256 patiënten in aanmerking komen. Expected cost per patient per year Cost € 85,000.00 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 drugs.com/price-guide/komzifti; accessdata.fda.gov/drugsatfda_docs/label/2025/220305s000lbl.pdf; KO-MEN-001 studie Additional remarks Er is geen Nederlandse lijstprijs bekend. In de Verenigde Staten kost een verpakking van 90 capsules (200mg) $47.382,70. Uitgaande van een dosering van 600mg, kost één toediening $1.579,42. Bij een dagelijkse toediening en behandelduur van 2,2 maanden, komen de totale behandelkosten per patiënt uit op ongeveer $100.000 (ofwel €85.000). Potential total cost per year Total cost € 21,760,000.00 Total cost for sluice Additional remarks Off label use Off label use Yes Indications off label use Kinderleukemie References Expertopinie Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks