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Luspatercept

Reblozyl

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

Registration

Registration route Centralised (EMA)
Submission date May 2019
Expected Registration June 2020
Orphan drug Yes
Registration phase Registration application pending
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
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

Expected patient volume per year

Patient volume

< 180

Market share is generally not included unless otherwise stated.
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

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 (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

Other information

There is currently no futher information available.