Extended indication Idiopathic thrombocytopenic purpura (ITP)
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Efgartigimod
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialit├ę)
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

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date August 2021
Expected Registration September 2022
Orphan drug No
Registration phase Registration application pending

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

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 Myasthenia gravis; Pemphigus vulgaris
References Adis Insight

Other information

There is currently no futher information available.