Fostamatinib Medicine 13 December 2018 Extended indication Tavlesse is indicated for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. Therapeutic value No judgement Total cost € 8,925,000.00 Registration phase Positive CHMP opinion Product Active substance Fostamatinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Other hematology Extended indication Tavlesse is indicated for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. Current proprietary name Already available biosimilars / generics Proprietary name Tavalisse Manufacturer Rigel Portfolio holder Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Prodrug voor het actieve bestandsdeel tamatinib wat een remmer is van de tyrosinekinase Syk. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date October 2018 Expected Registration January 2020 Orphan drug No Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in november 2019. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation 56% van de patiënten met een stabiele respons behielden die voor ten minste 24 maanden. Duration of treatment Median 8.9 continuous Frequency of administration 2 times a day Dosage per administration 100 mg References Bussel et al. Am J Hematol. 2018 Apr 26; ASH2018, paper No: 736 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 Additional remarks ITP komt bij ongeveer 1 tot 4 op de 100.000 mensen voor. Expected cost per patient per year Cost € 13,000.00 - 29,000.00 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 GIPdatabank Additional remarks Verwacht wordt dat de prijs in lijn ligt met de huidige therapiën. Eltrombopag kostte in 2016 €13.139,00 per patiënt per jaar. Romiplostim kostte in 2016 €28.998,00 per patiënt per jaar. Potential total cost per year Total cost € 8,925,000.00 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 Phase II study results published: Warm antibody autoimmune hemolytic anemia (wAIHA) References Additional remarks Other information Additional remarks