Extended indication

Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refra

Therapeutic value

No judgement

Registration phase

Clinical trials

Product

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 remarks
Fabrikant: Geron Corp. A first-in-class telomerase inhibitor.

Registration

Registration route

Centralised (EMA)

Submission date

2019

Expected Registration

2020

Orphan drug

No

Registration phase

Clinical trials

Additional remarks
Weesgeneesmiddel in de US niet in EU.

Therapeutic value

Current treatment options

RBC transfusies

Therapeutic value

No judgement

Frequency of administration

1 times every 4 weeks

Dosage per administration

7,5 mg/kg

References
NCT02598661

Expected patient volume per year

Patient volume

< 737

Market share is generally not included unless otherwise stated.

References
NKR
Additional remarks
Aantal diagnoses myelodysplastisch syndroom in 2016: 737.

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.