Imetelstat Active substance ImetelstatDomain Oncology and HematologyReason of inclusion in Horizonscan Geneesmiddelen New medicine (specialité)Main indication AML / MDSExtended indication Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refractory to prior therapy (refractory to ESA). 1. Product Manufacturer GeronMechanism of action Enzyme inhibitorRoute of administration IntravenousTherapeutical formulation Intravenous dripBudgetting framework Intermural (MSZ)Additional comments Fabrikant: Geron Corp. A first-in-class telomerase inhibitor. 2. Registration Registration route Centralised (EMA)Submission date 2019Expected Registration 2020Orphan drug NoRegistration phase Clinical trialsAdditional comments Weesgeneesmiddel in de US niet in EU. 3. Therapeutic value Current treatment options RBC transfusiesTherapeutic value No judgementDuration of treatment Not foundFrequency of administration 1 times every 4 weeksDosage per administration 7,5 mg/kgReferences NCT02598661 4. Expected patient volume per year Patient volume < 737 Market share is generally not included unless otherwise stated. References NKRAdditional comments Aantal diagnoses myelodysplastisch syndroom in 2016: 737. 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 YesIndication extensions Primary or secondary myelofibrosis.References SPS; ASH2018, paper no: 685 9. Other information There is currently no futher information available.