Extended indication Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refra
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Imetelstat
Domain 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)
ATMP No
Submission date September 2023
Expected Registration October 2024
Orphan drug Yes
Registration phase Registration application pending
Additional remarks Primary completion datum in augustus 2022. Inmiddels ingediend bij de FDA.

Therapeutic value

Current treatment options RBC transfusies
Therapeutic value No estimate possible yet
Frequency of administration 1 times every 4 weeks
Dosage per administration 7,5 mg/kg
References NCT02598661

Expected patient volume per year

Patient volume

< 361

Market share is generally not included unless otherwise stated.

References NKR2021(1); Expert opinie(2);
Additional remarks Aantal diagnoses myelodysplastisch syndroom in 2021 722 (1). Ongeveer de helft krijgt te maken met een relapse 361 (2).

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.