Rilzabrutinib Medicine 03 December 2024 Extended indication Voor de behandeling van trombocytopenie bij volwassenen en adolescenten met persistente of chronische immuun gemedieerde trombocytopenische purpura (ITP) Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Rilzabrutinib Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Voor de behandeling van trombocytopenie bij volwassenen en adolescenten met persistente of chronische immuun gemedieerde trombocytopenische purpura (ITP) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Sanofi Portfolio holder Sanofi Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Film-coated tablet Budgetting framework Extramural (GVS) Centre of expertise UMC Utrecht, LUMC, Hagaziekenhuis, UMCG Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date October 2024 Expected Registration November 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Fabrikant verwacht registratie het vierde kwartaal van 2025. Therapeutic value Current treatment options Human normal immunoglobulin, Nplate (romiplostim), Revolade (eltrombopag), Tavlesse (fostamatinib) and certain corticosteroids. Some patients required surgery (splenectomy) to remove the spleen Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Nog geen fase 3-studieresultaten bekend. Duration of treatment Frequency of administration 2 times a day Dosage per administration 400 mg References Kuter, et al. N Engl J Med. 2022 (1); Kuter et al. Blood Adv 2024 (2); NCT04562766 (LUNA 3). Additional remarks Expected patient volume per year Patient volume 32 - 75 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References ITP Expertisenetwerk (1); Kuter et al. Blood Adv. 2024 (2); Ministerie van VWS. Kerncijfers bevolking (3); Trinity RWE Claims Analysis (2020) using IBM MarketScan and CMS FFS 5% sample from 2016-2018, projected to US population using 2018 US census data (https://www.census.gov/); n (primary + secondary ITP) = 8,544 (4); Trinity RWE Line of Therapy Analysis (2020) using IBM MarketScan Commercial claims (for patients <65) and Medicare Supplemental data (for patients >=65) from 2017-2018; n = 1,716 (5). Additional remarks Er zijn 1.450-3.350 prevalente patiënten in Nederland (2). In de Nederlandse praktijk zullen patiënten refractair op immunomodulerende medicatie en na refractair te zijn op minimaal 1 TPO-RA in aanmerking komen voor behandeling met rilzabrutinib. Amerikaanse Trinity Health data laten zien dat van de ITP populatie 25% hiervoor wordt behandeld, waarvan 9% vervolgens refractair is op TPO-RAs en in aanmerking zouden komen voor behandeling met rilzabrutinib. Toepassing van de percentages op de Nederlandse populatie komt uit op 32 tot 75 patiënten (4,5). 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 No Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks