Pevonedistat Medicine 08 June 2021 Extended indication 1st line High Risk Myelodysplastic Syndrome (HR-MDS) or low blast AML or non proliferative CMML. Therapeutic value No estimate possible yet Total cost Registration phase No registration expected Product Active substance Pevonedistat Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication 1st line High Risk Myelodysplastic Syndrome (HR-MDS) or low blast AML or non proliferative CMML. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Takeda Portfolio holder Mechanism of action Other Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks NEDD8 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date December 2021 Expected Registration 2023 Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks De Fase 3 PANTHER (Pevonedistat-3001) studie bereikte geen vooraf gedefinieerde statistische significantie voor het primaire eindpunt van event-free survival. Momenteel wordt er geen registratie verwacht voor Pevonedistat Therapeutic value Current treatment options Azacitadine Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times every 4 weeks Dosage per administration 20 mg/m2 References PANTHER (NCT03268954) Additional remarks Expected patient volume per year Patient volume < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks In 2018 waren er 728 diagnoses MDS. Hiervan is een klein gedeelte hoog risico. Er wordt verwacht dat er maximaal 100 patiënten voor in aanmerking zullen komen. Expected cost per patient per year Cost References Fabrikant Additional remarks Nog niet bekend Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions In fase 3: Acute myeloid leukaemia; Chronic myelomonocytic leukaemia References Fabrikant; Adis insight Additional remarks Verwachte indicatie uitbreiding is in 2025. Other information Additional remarks