Luspatercept Medicine 13 December 2018 Extended indication Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy and for the treatment of adult patients with transfusion-dependent anaemia associated with beta-thalassaemia. Therapeutic value No judgement Total cost € 3,073,520.00 Registration phase Registered Product Active substance Luspatercept Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy and for the treatment of adult patients with transfusion-dependent anaemia associated with beta-thalassaemia. Current proprietary name Already available biosimilars / generics Proprietary name Reblozyl Manufacturer Celgene Portfolio holder Mechanism of action Other Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Erythroid Maturation Agent Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date May 2019 Expected Registration June 2020 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in april 2020. Therapeutic value Current treatment options RBC transfusies, allogene stamceltransplantatie. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Luspatercept zorgt mogelijk voor transfusieonafhankelijkheid. Duration of treatment Median 49 week / weeks Frequency of administration 1 times every 3 weeks Dosage per administration 1.0 mg/kg - 1.75 mg/kg References MEDALIST: NCT02631070 Additional remarks Medalist gepubliceerd in NEJM Jan 2020. Expected patient volume per year Patient volume 40 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR, expert opinie, https://hematologiegroningen.nl/patienten/content/3MDS.htm. Additional remarks Aantal diagnoses myelodysplastisch syndroom in 2016: 737. Hiervan is naar schatting zo'n 5% van de patiënten lager-risico MDS patiënt met ringsideroblasten en onvoldoende response op/relapse na epo en transfusie-afhankelijk (ongeveer 40 patiënten). Mogelijk wordt deze inschatting iets hoger als we de SF3b1 mutatie in acht nemen, maar de verwachting is dat dat geen enorme toename met zich mee zal brengen. Expected cost per patient per year Cost € 76,838.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Fabrikant; Z-index Additional remarks Per flacon 25mg: €1.478. Per flacon 75mg: €4.433. Voor een persoon van 70 kilo betekent dit 52/3=17,33*4.433= €76.838 voor de behandeling van een jaar. Potential total cost per year Total cost € 3,073,520.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions Myelofibrosis References adisinsight Additional remarks Op dit moment geen nieuwe uitbreidingen buiten de indicaties die op de Horizonscan te vinden zijn. Other information Additional remarks