Sebetralstat Medicine 05 December 2023 Extended indication Ekterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. Therapeutic value Possible benefit in ease of use Total cost € 49,776,000.00 Registration phase Registered Product Active substance Sebetralstat Domain Chronic immune diseases Reason of inclusion New medicine (specialité) Main indication Other chronic immune diseases Extended indication Ekterly is indicated for symptomatic treatment of acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. Current proprietary name Already available biosimilars / generics Proprietary name Ekterly Manufacturer Kalvista Portfolio holder Mechanism of action Enzyme inhibitor Route of administration Oral Therapeutical formulation Dispersible tablet Budgetting framework Extramural (GVS) Centre of expertise Amsterdam UMC Additional remarks Sebetralstat is het eerste orale geneesmiddel om aanvallen van hereditair angio-oedeem te behandelen. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New therapeutical formulation ATMP No Submission date August 2024 Expected Registration September 2025 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in juli 2025. Therapeutic value Current treatment options Icatibant (Firazyr), uit plasma verkregen C1-esteraseremmerconcentraat (Cinryze, Berinert), recombinant C1-esteraseremmerconcentraat (Conestat alfa, Ruconest). Therapeutic value Possible benefit in ease of use This assessment does not indicate any potential inclusion in the package. Substantiation De huidige therapeutische opties vereisen parenterale toediening. Sebetralstat wordt oraal toegediend. Duration of treatment Frequency of administration Dosage per administration 600mg zo nodig References NCT05259917; Aygoren-Pursun et al. Lancet. 2023; Marc et al. NEJM. 2024. 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 is. Expected cost per patient per year Cost € < 146,400.00 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 Zorginstituut Nederland. 2025. Additional remarks De kosten zullen mogelijk gelijk zijn aan berotralstat (Orladeyo): €146.365. Mogelijk zullen de kosten lager zijn door het on-demand gebruik van sebetralstat. Potential total cost per year Total cost € 49,776,000.00 Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks