Decitabine / cedazuridine Medicine 04 June 2024 Extended indication Extension of indication to include treatment of adult patients with myelodysplastic syndromes (MDS) and treatment of adult patients with chronic myelomonocytic leukaemia (CMML). Therapeutic value Possibly no place in the treatment regimen Total cost Registration phase No registration expected Product Active substance Decitabine / cedazuridine Domain Hematology Reason of inclusion Indication extension IND Main indication AML / MDS Extended indication Extension of indication to include treatment of adult patients with myelodysplastic syndromes (MDS) and treatment of adult patients with chronic myelomonocytic leukaemia (CMML). Current proprietary name Already available biosimilars / generics Proprietary name Inaqovi Manufacturer Otsuka Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Unknown Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-11-14-november-2024. Therapeutic value Current treatment options Therapeutic value Possibly no place in the treatment regimen This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Expertopinie Additional remarks Decitibine is niet geregistreerd voor deze indicatie (intraveneus). Een vergelijkende studie ontbreekt. Expected patient volume per year Patient volume < 0 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR2021; Hematon; expertopinie Additional remarks De incidentie van MDS bedraagt ongeveer 800 patiënten in Nederland. In Nederland krijgen elk jaar ongeveer 100 mensen CMML. De werkgroep schat in dat het middel niet wordt ingezet en er geen patiënten met decitabine/cedazuridine worden behandeld. 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks Otsuka has withdrawn the application for the extension of indication (treatment of adult patients with myelodysplastic syndromes (MDS) and treatment of adult patients with chronic myelomonocytic leukaemia (CMML)).