Active substance Imetelstat
Domain Oncology and Hematology
Reason of inclusion New medicine (specialité)
Main indication AML / MDS
Extended indication Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refractory to prior therapy (refractory to ESA).


Manufacturer Geron
Mechanism of action Enzyme inhibitor
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Fabrikant: Geron Corp. A first-in-class telomerase inhibitor.


Registration route Centralised (EMA)
Submission date 2021
Expected Registration 2022
Orphan drug No
Registration phase Clinical trials
Additional comments Weesgeneesmiddel in de Verenigde Staten niet in Europa.

Therapeutic value

Current treatment options RBC transfusies
Therapeutic value No judgement
Duration of treatment Not found
Frequency of administration 1 times every 4 weeks
Dosage per administration 7,5 mg/kg
References NCT02598661

Expected patient volume per year

Patient volume

< 369

Market share is generally not included unless otherwise stated.

References NKR
Additional comments Aantal diagnoses myelodysplastisch syndroom in 2016: 737. Ongeveer de helft krijgt te maken met een relapse (369).

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Primary or secondary myelofibrosis.
References SPS; ASH2018, paper no: 685

Other information

There is currently no futher information available.