Eltrombopag Medicine 03 December 2024 Extended indication Extension of indication to include second-line treatment of paediatric patients aged 2 years and above with acquired severe aplastic anaemia (SAA) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Eltrombopag Domain Cardiovascular diseases Reason of inclusion Indication extension IND Main indication Other non-oncological hematological medications Extended indication Extension of indication to include second-line treatment of paediatric patients aged 2 years and above with acquired severe aplastic anaemia (SAA) Current proprietary name Already available biosimilars / generics Proprietary name Revolade Manufacturer Novartis Portfolio holder Mechanism of action Receptor agonist Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Thrombopoietin receptor agonist Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date September 2024 Expected Registration July 2025 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van EMA-indiening in september 2024 en een gemiddelde doorlooptijd van 10 maanden voor een indicatieuitbreiding. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT03025698 (CETB115E2201) Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Horizonscan Geneesmiddelen eltrombopag (1) Additional remarks De incidentie bedraagt ongeveer 2:1.000.000, dit zijn ongeveer 36 Nederlandse patiënten per jaar (1). Het patiëntvolume dat in aanmerking komt voor deze pediatrische indicatieuitbreiding zal gering zijn. Expected cost per patient per year Cost € < 39,500.00 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. References Horizonscan Geneesmiddelen eltrombopag (1) 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 Unknown Indication extensions References Additional remarks Other information Additional remarks