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Luspatercept

Reblozyl

Extended indication Beta-thalassemia, transfusion dependent
Therapeutic value No judgement
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
Proprietary name Reblozyl
Manufacturer celgene
Mechanism of action Other
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
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 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
Substantiation Leidt mogelijk tot reductie in RBC transfusies of transfusieonafhankelijkheid.
Duration of treatment continuous
Frequency of administration 1 times every 3 weeks
Dosage per administration 1 mg/kg
References NCT02604433

Expected patient volume per year

Patient volume

< 150

Market share is generally not included unless otherwise stated.
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

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 myelodysplastic syndromes
References clinicaltrials.gov

Other information

There is currently no futher information available.