Masitinib Medicine 03 June 2025 Extended indication Masitinib in combination with best supportive care for third line or later treatment of severe Indolent systemic mastocytosis and of for the treatment of severe smouldering Systemic mastocytosis i in adults and elderly who are unresponsive to optimal symptomatic treatment. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Masitinib Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Masitinib in combination with best supportive care for third line or later treatment of severe Indolent systemic mastocytosis and of for the treatment of severe smouldering Systemic mastocytosis i in adults and elderly who are unresponsive to optimal symptomatic treatment. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer AB Science Portfolio holder Mechanism of action Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP No Submission date December 2025 Expected Registration January 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Tot op heden is er geen behandeling die de mestcellen kan doen verdwijnen. De therapie is dan ook vooral gericht op het kalmeren van de mestcellen en het wegvangen van de stoffen die zijn vrijgekomen. In de meeste gevallen worden zogenaamde histamine groep 1- en groep 2-blokkers voorgeschreven. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT04333108 Additional remarks Expected patient volume per year Patient volume 1,980 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Van Doormaal JJ et al. Prevalence of indolent systemic mastocytosis in a Dutch region. J Allergy Clin Immunol. 2013(1); Orphanet (2) Additional remarks Systemische mastocytose is een zeldzame ziekte die wordt veroorzaakt door een overmatige deling van afwijkende mestcellen. Door de grote variatie aan symptomen en de relatieve onbekendheid met de ziekte wordt de diagnose vaak laat gesteld. De prevalentie van systemische mastocytose is ongeveer 13 op de 100.000 Nederlanders. Dit betekent dat er zo’n 2.200 patiënten in Nederland zijn (1). Indolente SM (ISM) vertegenwoordigt 90% van alle gevallen van SM (2). Dit betekent dat er volgens de huidige indicatie 1.980 patiënten in aanmerking zullen komen in Nederland. 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