Extended indication Idiopathic thrombocytopenic purpura (ITP)
Therapeutic value No estimate possible yet
Total cost 72,765,100.00
Registration phase Clinical trials


Active substance Efgartigimod
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Idiopathic thrombocytopenic purpura (ITP)
Manufacturer Argenx
Mechanism of action Receptor antagonist
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)


Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date 2023
Expected Registration 2024
Orphan drug No
Registration phase Clinical trials
Additional comments Estimated Primary completion datum fase 3 studie in maart 2022.

Therapeutic value

Current treatment options Corticosteroïden, Immunoglobulinen, Eltrombopag.
Therapeutic value No estimate possible yet
Substantiation Met de huidige behandelopties zijn er al veel alternatieve behandelopties. Efgartigimod zal de concurrentie aan moeten gaan met de huidige behandelopties. Er is op dit moment nog geen directe vergelijking mogelijk.
Dosage per administration 10 mg/kg
References NCT04188379, NCT04225156

Expected patient volume per year

Patient volume

170 - 680

Market share is generally not included unless otherwise stated.

References erfelijkheid.nl, Inschatting Fostamatinib (Horizonscan Geneesmiddelen);
Additional comments ITP komt bij ongeveer 1 tot 4 op de 100.000 mensen voor. Dit betekent dat er omgerekend mogelijk 170 tot 680 mensen in aanmerking zouden komen voor deze behandeling.

Expected cost per patient per year

Cost < 171,212.00
References drugs.com
Additional comments Op basis van de prijs in de Verenigde Staten is de Nederlandse prijs ingeschat op €171.212,8. De prijs in de Verenigde Staten ligt vaak hoger dan de Nederlandse prijs.

Potential total cost per year

Total cost


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 Yes
Indication extensions Myasthenia gravis; Pemphigus vulgaris; ITP; ; Chronic inflammatory demyelinating polyneuropathy ; four new efgartigimod indications to be initiated in 2022: membranous nephropathy, lupus nephritis, Sjogren’s syndrome, COVID-19 mediated postural orthostatic tachycardia syndrome (POTS
References https://www.argenx.com/news/argenx-highlights-strategic-priorities-2022

Other information

There is currently no futher information available.