Parsaclisib Medicine 07 June 2022 Extended indication Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL). Therapeutic value No estimate possible yet Total cost Registration phase No registration expected Product Active substance Parsaclisib Domain Hematology Reason of inclusion New medicine (specialité) Main indication Indolent non-Hodgkin’s lymphoma Extended indication Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL). Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Incyte Portfolio holder Incyte Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: Antineoplastic agents, phosphatidylinositol-3-kinase (PI3K) inhibitors Registration Registration route Centralised (EMA) Type of trajectory Conditional marketing authorisation Particularity Unknown ATMP No Submission date Expected Registration Orphan drug Yes Registration phase No registration expected Reimbursement Medicine sluice Additional remarks Incyte heeft eind juli 2022 de ‘Marketing Authorization Application’ (MAA) voor parsaclisib voor marginal zone lymphoma (MZL) ingetrokken bij de EMA. Parsaclisib zal dus niet geregistreerd worden voor MZL. Therapeutic value Current treatment options Andere PI3K delta remmers zoals, umbralisib en copalisib. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Uit de HOVON-richtlijn 2021 staat dat waar mogelijk patiënten met R/R MZL geïncludeerd moeten worden in studies, bijvoorbeeld met nieuwe vormen van immuuntherapie of ‘targeted drugs’. Er zijn nog geen gepubliceerde data uit de studie Ferero bekend. Duration of treatment Frequency of administration 1 times a day Dosage per administration 20 mg eerste 8 weken, daarna 2,5 mg References HOVON-richtlijn, Marginale Zone Lymfoom (2021) NCT03144674 (CITADEL-204, fase 2b) NCT04796922 (CITADEL-302, fase 3 icm folliculair lymfoom) Additional remarks Expected patient volume per year Patient volume < 225 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References HOVON-richtlijn MZL (2021) Horizonscan record umbralisib Additional remarks Ongeveer 10-15% van de Non Hodgkin Lymfoom gevallen is een marginale zone lymfoom. In Nederland zijn er 3.000 nieuwe patiënten per jaar. Hiervan zijn dus ongeveer 300 patiënten met marginale zone lymfoom. Naar verwachting zal 50% hiervan op enig moment een tweedelijnsbehandeling ondergaan. Het patiëntvolume zal daardoor maximaal 150-225 patiënten bedragen. Echter worden er beperkte patiëntaantallen verwacht gezien concurrerende geneesmiddelen. Expected cost per patient per year Cost References medicijnkosten.nl Additional remarks Kosten per patiënt per jaar afhankelijk van ziekteprogressie of onaanvaardbare toxiciteit. Kosten idelalisib (Zydelig) €125 per dag (op basis van: 2xper dag) 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 Yes Indication extensions Mantle Cell Lymphoma & Follicular lymphoma References Mantle Cell Lymphoma (NCT03235544; fase 2 , NCT04849715; fase 3), Follicular lymphoma (NCT03126019; fase 2 & NCT04796922; fase 3 icm R/R MZL) Additional remarks CITADEL-203 (NCT03126019) is evaluating patients with relapsed or refractory follicular lymphoma (FL) Grade 1, 2 or 3a who received at least two prior systemic therapies, had an Eastern Cooperative Oncology Group performance status (ECOG PS) ≤2, and were ineligible for hematopoietic stem cell transplantation (HSCT). CITADEL-205 (NCT03235544) is evaluating patients with relapsed or refractory mantle cell lymphoma (MCL), who received one to three prior systemic therapies and were either naive to or were previously treated with a BTK inhibitor. Eligible patients had an ECOG PS ≤2, and radiologically measurable lymphadenopathy or extranodal lymphoid malignancy. Other information Additional remarks