Bezuclastinib Medicine 04 June 2026 Extended indication Bezuclastinib in combination with best supportive care for treatment of moderate to severe nonadvanced systemic mastocytosis in adults and elderly whose symptoms are not adequately controlled. Therapeutic value Possible added value Total cost Registration phase Clinical trials Product Active substance Bezuclastinib Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Bezuclastinib in combination with best supportive care for treatment of moderate to severe nonadvanced systemic mastocytosis in adults and elderly whose symptoms are not adequately controlled. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Cogent Portfolio holder Cogent Mechanism of action Tyrosine kinase inhibitor Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity ATMP Submission date June 2026 Expected Registration July 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 Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Er is een aantoonbaar voordeel gevonden in de trial. Duration of treatment Frequency of administration Dosage per administration References NCT05186753 (SUMMIT); Expertopinie Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Van Doormaal JJ et al. J Allergy Clin Immunol. 2013 (1) 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). Expected cost per patient per year Cost References Additional remarks Er is nog niets bekend over de mogelijke prijs. 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