Sebetralstat Medicine 05 December 2023 Extended indication Indicated for the treatment of patient-recognised Hereditary Angioedema attacks in adults and pedriatic patients 12 years and older Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Sebetralstat Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Indicated for the treatment of patient-recognised Hereditary Angioedema attacks in adults and pedriatic patients 12 years and older Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Kalvista Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Film-coated tablet Budgetting framework Extramural (GVS) Centre of expertise Amsterdam UMC, location AMC, Amsterdam Additional remarks Sebetralstat will be the first oral on demand treatment for HAE and is currently in PhIII. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date June 2024 Expected Registration June 2025 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Bradykinin B2 receptor antagonist Firazyr C1 esterase inhibitor Berinert C1 esterase inhibitor Ruconest 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 600 mg References Aygoren-Pursun E, Zanichelli A, Cohn DM, et al. An investigational oral plasma kallikrein inhibitor for on-demand treatment of hereditary angioedema: a two-part, randomised, double-blind, placebo-controlled, crossover phase 2 trial. Lancet. Feb 11 2023;401(10375):458-469. doi:10.1016/S0140-6736(22)02406-0 Additional remarks on demand treatment of acute attacks Expected patient volume per year Patient volume < 340 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References https://www.worldometers.info/world-population/netherlands-population/ Ghazi A, Grant JA. Hereditary angioedema: epidemiology, management, and role of icatibant. Biologics. 2013;7:103-13. doi:10.2147/BTT.S27566 Additional remarks Hereditair angio-oedeem heeft een prevalentie van 1 op de 50.000. Dit zou betekenen dat er maximaal 350 patiënten in Nederland in aanmerking komen voor deze behandeling. Het zal met name ingezet worden ter preventie. Dat maakt dat het patiëntaantal mogelijk lager gaat zijn. Expected cost per patient per year Cost References Additional remarks No information available yet Potential total cost per year Total cost Total cost for sluice Additional remarks No information available yet 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