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.

1. 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

2. 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.

3. 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

4. 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.

5. 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).

6. 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.

7. Off label use

There is currently nothing known about off label use.

8. 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.

9. Other information

There is currently no futher information available.