Iopofosine I 131 Medicine 04 June 2026 Extended indication Iopofosine i 131 monotherapy for third line or later treatment of relapsed or refractory Waldenstromʼs macroglobulinaemia in adults and elderly. Therapeutic value Total cost Registration phase Clinical trials Product Active substance Iopofosine I 131 Domain Hematology Reason of inclusion New medicine (specialité) Main indication Other hematology Extended indication Iopofosine i 131 monotherapy for third line or later treatment of relapsed or refractory Waldenstromʼs macroglobulinaemia in adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Cellectar Portfolio holder Cellectar Mechanism of action Other, see general comments Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Werkingsmechanisme: ionising radiation emitters Registration Registration route Centralised (EMA) Type of trajectory Particularity New medicine with Priority Medicines (PRIME) ATMP Submission date August 2026 Expected Registration September 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Therapeutic value Substantiation De CLOVER-WaM-studie toonde een algehele respons (ORR) van 80% na BTK remmer. Abstract ASH 2024 ; 65 patienten, 72% PFS na 18 maanden , langere follow-up data zijn er nog niet. Bijwerkingen: pancytopenie (13% infecties) voordeel dat het een eenmalige behandeling is en goede respons laat zien. Duration of treatment Frequency of administration Dosage per administration References 2 CLOVER-WaM trial (NCT02952508) Additional remarks Expected patient volume per year Patient volume < 470 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1: IKNL; 2: HOVON Lymfoom werkgroep. Richtlijn voor de diagnostiek, behandeling en follow-up van Waldenström’s Macroglobulinemie (WM) en IgM gerelateerde ziekte. 2020; Additional remarks In 2024 kregen 364 mensen de ziekte van Waldenström (1). In totaal leven er in Nederland meer dan 1.500 patiënten met deze ziekte. In 2024 waren er ongeveer 470 gebruikers van BTK-remmers voor de ziekte van Waldenström (2). Dit zal waarschijnlijk 2e of 3e lijn na covalente BTK remmer gegeven worden waardoor het patiëntvolume lager zal zijn. 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks