Rilzabrutinib Medicine 03 December 2024 Extended indication Wayrilz is indicated for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments Therapeutic value Possible equal value Total cost Registration phase Positive CHMP opinion Product Active substance Rilzabrutinib Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Wayrilz is indicated for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments Current proprietary name Already available biosimilars / generics Proprietary name Wayrilz 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 December 2025 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in oktober 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 Possible equal value This assessment does not indicate any potential inclusion in the package. Substantiation In de RCT van rilzabrutinib versus placebo werd een overall respons van 64% versus 32% gevonden. Op het primaire eindpunt (durable response) 23% versus 0%. Het is lastig om een uitspraak te doen aangezien het tegen placebo getest is. Er wordt een gelijke waarde ten opzichte van fostamatinib verwacht. Duration of treatment Frequency of administration 2 times a day Dosage per administration 400mg References Kuter, et al. N Engl J Med. 2022 (1); Kuter et al. Blood Adv 2024 (2); NCT04562766 (LUNA 3). Kuter, Blood maart 2025; DOI: 10.1182/blood.2024027336. 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 tot 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 Er is nog niets bekend over de mogelijk kosten in Nederland. 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