Efgartigimod Medicine 08 June 2021 Extended indication Idiopathic thrombocytopenic purpura (ITP) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product 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) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Argenx Portfolio holder Mechanism of action Receptor antagonist Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date August 2021 Expected Registration September 2022 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Corticosteroïden, Immunoglobulinen, Eltrombopag. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. 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. Duration of treatment Frequency of administration Dosage per administration 10 mg/kg References NCT04188379, NCT04225156 Additional remarks Expected patient volume per year Patient volume 170 - 680 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References erfelijkheid.nl, Inschatting Fostamatinib (Horizonscan Geneesmiddelen); Additional remarks 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 References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Myasthenia gravis; Pemphigus vulgaris References Adis Insight Additional remarks Other information Additional remarks