Luspatercept

Active substance Luspatercept
Domain Oncology and Hematology
Reason of inclusion in Horizonscan Geneesmiddelen New medicine (specialité)
Main indication AML / MDS
Extended indication Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy and for the treatment of adult patients with transfusion-dependent anaemia associated with beta-thalassaemia.

Product

Proprietary name Reblozyl
Manufacturer Celgene
Mechanism of action Other
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Additional comments Erythroid Maturation Agent

Registration

Registration route Centralised (EMA)
Submission date May 2019
Expected Registration June 2020
Orphan drug Yes
Registration phase Positive CHMP opinion
Additional comments Positieve CHMP-opinie in april 2020.

Therapeutic value

Current treatment options RBC transfusies, allogene stamceltransplantatie
Therapeutic value No judgement
Substantiation Luspatercept zorgt mogelijk voor transfusieonafhankelijkheid
Duration of treatment Median 49 week / weeks
Frequency of administration 1 times every 3 weeks
Dosage per administration 1.0 mg/kg - 1.75 mg/kg
References MEDALIST: NCT02631070
Additional comments Medalist gepubliceerd in NEJM Jan 2020

Expected patient volume per year

Patient volume

40

Market share is generally not included unless otherwise stated.

References NKR, expert opinie, https://hematologiegroningen.nl/patienten/content/3MDS.htm.
Additional comments Aantal diagnoses myelodysplastisch syndroom in 2016: 737. Hiervan is naar schatting zo'n 5% van de patiënten lager-risico MDS patiënt met ringsideroblasten en onvoldoende response op/relapse na epo en transfusie-afhankelijk (ongeveer 40 patiënten). Mogelijk wordt deze inschatting iets hoger als we de SF3b1 mutatie in acht nemen, maar de verwachting is dat dat geen enorme toename met zich mee zal brengen.

Expected cost per patient per year

Cost < 160,000
References https://www.biopharmadive.com/news/celgene-win-fda-approval-luspatercept-reblozyl-beta-thalassemia/566968/
Additional comments In de Verenigde Staten is de prijs voor 25 mg Reblozyl gezet op $3.441. Voor een persoon van 70 kg en een behandeling van een jaar komt dit neer op 52 flacons en dus $178.932 (~€160.000).

Potential total cost per year

Total cost

6,400,000

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References adisinsight
Additional comments Op dit moment geen nieuwe uitbreidingen buiten de indicaties die op de Horizonscan te vinden zijn.

Other information

There is currently no futher information available.