Extended indication Lower risk myelodysplastic syndrome (MDS) RS+, refractory or ineligible to ESA and regularly transfused
Therapeutic value No judgement
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
Proprietary name Reblozyl
Manufacturer celgene
Route of administration Subcutaneous
Therapeutical formulation Injection
Additional remarks Erythroid Maturation Agent

Registration

Registration route Centralised (EMA)
Submission date January 2019
Expected Registration February 2020
Orphan drug Yes
Registration phase Clinical trials
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
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

Expected patient volume per year

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

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension Yes
Indication extensions Phase III Beta-thalassaemia
References clinicaltrials.gov

Other information

There is currently no futher information available.