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Sebetralstat

Extended indication Indicated for the treatment of patient-recognised Hereditary Angioedema attacks in adults and pedria
Therapeutic value No estimate possible yet
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
Manufacturer Kalvista
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

Therapeutic value

Current treatment options Bradykinin B2 receptor antagonist Firazyr C1 esterase inhibitor Berinert C1 esterase inhibitor Ruconest
Therapeutic value No estimate possible yet
Duration of treatment lifetime
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.
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

Additional remarks No information available yet

Potential total cost per year

Additional remarks No information available yet

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.