Extended indication

Treatment of adult patients with transfusion-dependent anaemia due to very low, low and intermediate

Therapeutic value

No judgement

Total cost

6,400,000.00

Registration phase

Positive CHMP opinion

Product

Active substance

Luspatercept

Domain

Oncology and Hematology

Reason of inclusion

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.

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

< 160,000.00

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

6,400,000.00

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.