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 Clinical trials Product Active substance Luspatercept Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Leukemia 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 Unknown 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 January 2019 Expected Registration February 2020 Orphan drug Yes Registration phase Clinical trials 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 EMA: "At the time of designation, some medicines were authorised in the EU for the treatment of MDSs. The choice of treatment for MDSs depended on a number of factors, including the type and the extent of the disease, whether it had been treated before, and the patient’s age, symptoms and general state of health. The main treatments for MDSs included chemotherapy (medicines to treat cancer) and bone marrow transplantation. This is a complex procedure where the bone marrow of the patient is cleared of cells and replaced with healthy bone marrow cells from a matched donor." RBS 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 References NCT02631070, EMA Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References EMA, Hematon Additional remarks EMA: "At the time of designation, myelodysplastic syndromes affected approximately 2 in 10,000 people in the European Union (EU)." Dit komt neer op maximaal 3.400 patiënten in Nederland. Hematon: "MDS is een zeldzame ziekte die vooral bij mensen tussen de zestig en negentig jaar voorkomt. Het wordt vaker bij mannen geconstateerd. In Nederland gaat het om ongeveer zevenhonderd nieuwe gevallen per jaar." 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 References clinicaltrials.gov Additional remarks Other information Additional remarks