Lonvoguran ziclumeran Medicine 04 December 2025 Extended indication Lonvoguran ziclumeran monotherapy for treatment of type I or II hereditary angioedema in adolescents over 16 years of age, adults and elderly. Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Lonvoguran ziclumeran Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Lonvoguran ziclumeran monotherapy for treatment of type I or II hereditary angioedema in adolescents over 16 years of age, adults and elderly. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Intellia Portfolio holder Intellia Mechanism of action Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks CRISPR-gentherapie. Registration Registration route Centralised (EMA) Type of trajectory Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date July 2026 Expected Registration August 2027 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Tijdslijn op basis van IHSI-inschatting. Therapeutic value Current treatment options Acute behandeling: C1-esteraseremmer, plasma-derived (Berinert, Cinryze) en recombinant (Ruconest). Bradykinine B2-receptorantagonist (icatibant, Firazyr). Langetermijn/routineprofylaxe: C1-esteraseremmer (Cinryze). Kallikreïneremmer (lanadelumab [Takhzyro], berotralstat [Orladeyo]). Garadacimab is recent goedgekeurd. Pre-procedurele profylaxe: C1-esteraseremmer, plasma-derived (Berinert, Cinryze). Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De fabrikant meldt positieve driejarige resultaten. Bij tien patiënten verminderde het aantal aanvallen met gemiddeld 98%. Alle deelnemers bleven aanvalsvrij en zonder extra behandeling gedurende een mediane periode van 23 maanden. De therapie werd goed verdragen en vertoonde een gunstig veiligheidsprofiel. De wereldwijde fase 3-studie, HAELO, is nog niet afgerond, waardoor een inschatting van de therapeutische waarde nog niet is te maken. Duration of treatment Frequency of administration Dosage per administration 50mg References NCT06634420 (HAELO); Persbericht Intellia Therapeutics. 2025; Expertopinie. Additional remarks Expected patient volume per year Patient volume < 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GVS advies berotralstat (Orladeyo®) voor hereditair angio-oedeem. Zorginstituut Nederland. 2025 (1); Expertopinie (2) Additional remarks 230 patiënten worden medicamenteus behandeld voor hereditair angio-oedeem, waarvan 64 routine profylaxe (1). De inschatting is echter dat lonvo-z ingezet zal worden bij minder dan 10 patiënten (2). Expected cost per patient per year Cost References Additional remarks Mogelijk zeer hoge kosten wegens PRIME-status. 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