Luspatercept Medicine 13 December 2018 Extended indication Lower risk myelodysplastic syndrome (MDS) RS+, refractory or ineligible to ESA and regularly transfused Therapeutic value No judgement Total cost Registration phase Registration application pending Product Active substance Luspatercept Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Lower risk myelodysplastic syndrome (MDS) RS+, refractory or ineligible to ESA and regularly transfused 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 Unknown 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 MEDALIST; "met primary and secondary endpoints". AdisInsight: "Celgene and Acceleron announces intention to submit NDA and MAA to US FDA and EMA for Myelodysplastic syndromes and Beta-thalassaemia in first half of 2019." Fabrikant verwacht indiening in H1 2019 en registratie in H1 2020. Therapeutic value Current treatment options RBC transfusies 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 13 month / months Frequency of administration 1 times every 3 weeks Dosage per administration 1.0 mg/kg - 1.75 mg/kg References MEDALIST: NCT02631070 Additional remarks Expected patient volume per year Patient volume < 180 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. Naar schatting heeft een kwart van deze patiënten ringsideroblasten: zo'n 180 patiënten. 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 Beta-thalassaemia (nu ook opgenomen in de Horizonscan), Phase III first line Luspatercept Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in ESA Naïve Subjects Who Require Red Blood Cell Transfusions (COMMANDS trial) References clinicaltrials.gov Additional remarks Other information Additional remarks