Sebetralstat

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
Registration phase	Registration application pending

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	August 2024
Expected Registration	September 2025
Orphan drug	Yes
Registration phase	Registration application pending

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.

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	Momenteel nog geen informatie beschikbaar

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.