Luspatercept Medicine 12 June 2019 Extended indication Beta-thalassemia, transfusion dependent Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Luspatercept Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Beta-thalassemia, transfusion dependent 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 Extramural (GVS) 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 Registration application pending Reimbursement Medicine sluice Additional remarks Fabrikant verwacht indiening bij de EMA in H1 2019 en goedkeuring in H1 2020. Therapeutic value Current treatment options Bloedtransfusies en ijzerchelatie. Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Leidt mogelijk tot reductie in RBC transfusies of transfusieonafhankelijkheid. Duration of treatment Frequency of administration 1 times every 3 weeks Dosage per administration 1 mg/kg References NCT02604433 Additional remarks Expected patient volume per year Patient volume < 150 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References hematologienederland.nl Additional remarks In Nederland zijn ongeveer 100-200 patiënten bekend. De ziekte komt evenveel voor bij mannen als bij vrouwen. Gezien het echter gaat om een ernstige vorm van Beta-thalassemia zal maar een deel van de patiënten in aanmerking komen voor luspatercept. 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 Phase III myelodysplastic syndromes References clinicaltrials.gov Additional remarks Other information Additional remarks