Extended indication 1st line High Risk Myelodysplastic Syndrome (HR-MDS) or low blast AML or non proliferative CMML.
Therapeutic value No estimate possible yet
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.
Manufacturer Takeda
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments NEDD8 inhibitor

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date December 2021
Expected Registration 2023
Orphan drug Yes
Registration phase No registration expected
Additional comments 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
Duration of treatment continuous
Frequency of administration 1 times every 4 weeks
Dosage per administration 20 mg/m2
References PANTHER (NCT03268954)

Expected patient volume per year

Patient volume

< 100

Market share is generally not included unless otherwise stated.

References NKR
Additional comments 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

References Fabrikant
Additional comments Nog niet bekend

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

Off label use No

Indication extension

Indication extension Yes
Indication extensions In fase 3: Acute myeloid leukaemia; Chronic myelomonocytic leukaemia
References Fabrikant; Adis insight
Additional comments Verwachte indicatie uitbreiding is in 2025.

Other information

There is currently no futher information available.