Horse anti-human T lymphocyte immunoglobulin Medicine 07 December 2021 Extended indication Atgam is indicated for use in adults and in children aged 2 years and older for the treatment of acquired moderate to severe aplastic anaemia of known or suspected immunologic aetiology as part of standard immunosuppressive therapy in patients who are unsuitable for bone marrow transplant. Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Horse anti-human T lymphocyte immunoglobulin Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Atgam is indicated for use in adults and in children aged 2 years and older for the treatment of acquired moderate to severe aplastic anaemia of known or suspected immunologic aetiology as part of standard immunosuppressive therapy in patients who are unsuitable for bone marrow transplant. Current proprietary name Already available biosimilars / generics Proprietary name ATgam Manufacturer Pfizer Portfolio holder Mechanism of action Immunostimulation Route of administration Intravenous Therapeutical formulation Concentrate for solution for infusion Budgetting framework Intermural (MSZ) Centre of expertise LUMC Additional remarks ATGAM bestaat uit een mix van polyclonale antistoffen die verkregen wordt door immunisatie van paarden. Registration Registration route Decentralised Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date January 2020 Expected Registration March 2022 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Geen vergelijkbare behandelopties voor >80% van de patienten met aplastische anemie. Voor een klein deel van de patiënten met aplastische anemie tot 40 jaar kan allogene stamceltransplantatie overwogen worden bij aanwezigheid van een gematchte sibling donor. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration 16 mg/kg/day over 10 days or 20 mg/kg/day over 8 days or 40 mg/kg/day over 4 days References Richtlijn verworven aplastische anemie, maart 2019: https://hematologienederland.nl/wp-content/uploads/2019/07/richtlijn_verworven_aplastische_anemie_2019_definitief.pdf ; Scheinberg P, et al. N EngI J Med 2011;365:430-8; Additional remarks Treatment duration: 4 to 10 days (dependent on dose per day) Total recommended dose is 160mg/kg Expected patient volume per year Patient volume 28 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Nederlandse Aplastische Anemie register; Tjon JM, et al. Br J Haematol 2018;180:459-62. Additional remarks Expected cost per patient per year Cost References Additional remarks De fabrikant geeft aan dat de prijsstelling niet voor het eerste kwartaal van 2022 beschikbaar zal zijn. 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 No Indication extensions References Additional remarks Other information Additional remarks