Efgartigimod Medicine 08 June 2021 Extended indication Idiopathic thrombocytopenic purpura (ITP) Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials 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 Unknown Particularity Unknown ATMP No Submission date 2021 Expected Registration 2022 Orphan drug No Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Verwachte indieningsdatum op basis van primary completion datum in juni 2021. 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); 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