Imetelstat Medicine 12 June 2018 Extended indication Myelodysplastic syndromes (MDS) low risk patients; transfusion-dependent and relapsed after or refractory to prior therapy (refractory to ESA). Therapeutic value No judgement Total cost 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). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Geron Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fabrikant: Geron Corp. A first-in-class telomerase inhibitor. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2020 Expected Registration 2021 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Weesgeneesmiddel in de US niet in EU. Therapeutic value Current treatment options RBC transfusies Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times every 4 weeks Dosage per administration 7,5 mg/kg References NCT02598661 Additional remarks Expected patient volume per year Patient volume < 369 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks Aantal diagnoses myelodysplastisch syndroom in 2016: 737. Ongeveer de helft krijgt te maken met een relapse (369). Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Primary or secondary myelofibrosis. References SPS; ASH2018, paper no: 685 Additional remarks Other information Additional remarks