Berotralstat Medicine 04 December 2025 Extended indication Berotralstat monotherapy for prophylactic treatment of hereditary angioedema in children aged 2 to 12. Therapeutic value No estimate possible yet Total cost € 1,463,650.00 Registration phase Registration application pending Product Active substance Berotralstat Domain Chronic immune diseases Reason of inclusion Indication extension IND Main indication Other chronic immune diseases Extended indication Berotralstat monotherapy for prophylactic treatment of hereditary angioedema in children aged 2 to 12. Current proprietary name Already available biosimilars / generics Proprietary name Orladeyo Manufacturer BioCryst Portfolio holder Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date May 2025 Expected Registration June 2026 Orphan drug No Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Verwachte registratie op basis van IHSI-inschatting. Therapeutic value Current treatment options Langetermijn/routineprofylaxe: C1-esteraseremmer (Cinryze). Kallikreïneremmer (lanadelumab [Takhzyro], berotralstat [Orladeyo]). Garadacimab is recent goedgekeurd. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 1 times a day Dosage per administration References NCT05453968 (APeX-P) 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; Expertopinie. Additional remarks 230 patiënten worden medicamenteus behandeld voor HAE, waarvan 64 routine profylaxe. In de budget impact analyse van berotralstat voor 12 jaar en ouder werd geschat dat er 31 patiënten gebruik zouden maken van berotralstat. Het patiëntvolume zal enigszins toenemen met deze indicatieuitbreiding naar twee jaar en ouder. Expected cost per patient per year Cost € < 146,365.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GVS advies berotralstat (Orladeyo®) voor hereditair angio-oedeem. Zorginstituut Nederland. 2025. Additional remarks De kosten zullen mogelijk lager uitvallen door een lagere dosering. Potential total cost per year Total cost € 1,463,650.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks