Trilaciclib

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	Clinical trials

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 remarks	NEDD8 inhibitor

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	December 2021
Expected Registration	April 2023
Orphan drug	Yes
Registration phase	Clinical trials
Additional remarks	De fabrikant verwacht registratie in april 2023.

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 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.

Patient volume

< 100

Market share is generally not included unless otherwise stated.

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

References	Fabrikant
Additional remarks	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 remarks	Verwachte indicatie uitbreiding is in 2025.

Other information

There is currently no futher information available.

Pevonedistat