Imetelstat

Active substance
Imetelstat
Domain
Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen
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).

1. Product

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.

2. Registration

Registration route
Centralised (EMA)
Submission date
2020
Expected Registration
2021
Orphan drug
No
Registration phase
Clinical trials
Additional comments
Weesgeneesmiddel in de US niet in EU.

3. 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

4. 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).

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

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

9. Other information

There is currently no futher information available.