Revumenib Medicine 03 December 2024 Extended indication MLLr/KMT2A Gene Rearrangement or NPM1 Mutation relapsed or refractory acute leukaemia Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Revumenib Domain Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication MLLr/KMT2A Gene Rearrangement or NPM1 Mutation relapsed or refractory acute leukaemia Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Syndax Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: Menin inhibitors; Protein-protein interaction inhibitors Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date June 2025 Expected Registration July 2026 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Azacitidine en venetoclax combinatie. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Er lopen op dit moment twee fase 2 studies, waarbij revumenib vergeleken wordt met de standard of care. Revumenib is een menin inhibitor. Er wordt verwacht dat er nog meer menin inhibitors zullen volgen en deze een rol gaan spelen bij de behandeling van leukemie. Duration of treatment Frequency of administration Dosage per administration References NCT04065399 (AUGMENT-101) Additional remarks Expected patient volume per year Patient volume < 43 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References (1) NKR2021; (2) Arlene Redner, Rachel Kessel, in Lanzkowsky's Manual of Pediatric Hematology and Oncology (Seventh Edition), 2022 Additional remarks Er waren 853 AML diagnoses in 2021 (1). Mutaties van het KMT2A (MLL) gen bij 11q23. 3 wordt gezien in 4% tot 5% in alle leeftijdsgroepen van de novo volwassenen met AML echter in een veel grotere mate in jongeren (tot 22%) en met name kinderen (2). Er komen maximaal 43 patiënten in aanmerking. Aangezien vooral ouderen behandeld zullen worden met dit geneesmiddel zal het uiteindelijke aantal waarschijnlijk lager liggen. Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost 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