Imetelstat

Extended indication	Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refractory to prior therapy (refractory to ESA).
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 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.

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

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).

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).

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

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

There is currently no futher information available.