Active substance
Chronic immune diseases
Reason of inclusion in Horizonscan Geneesmiddelen
New medicine (specialité)
Main indication
Other chronic immune diseases
Extended indication
Treatment for the Prevention of Attacks in patients with Type I and II Hereditary Angioedema

1. Product

Mechanism of action
Route of administration
Therapeutical formulation
Budgetting framework
Extramural (GVS)
Additional comments
Bradykinine remmer

2. Registration

Registration route
Centralised (EMA)
Submission date
April 2020
Expected Registration
May 2021
Orphan drug
Registration phase
Registration application pending
Additional comments
Datum indiening en verwachte registratie op basis van actual primary completion date van fase 3 studie in April 2019.

3. Therapeutic value

Current treatment options
C1-esteraseremmers en icatibant en Cinryze. Daarnaast ook de monoklonale antistof lanadelumab.
Therapeutic value
No judgement
De verwachting is dat berotralstat niet effectiever zal zijn dan lanadelumab.
Duration of treatment
Not found
Frequency of administration
1 times a day
Dosage per administration
150 mg

4. Expected patient volume per year

Additional comments
Hereditair angio-oedeem heeft een prevalentie van 1-9 / 100.000. Dit zou betekenen dat 170 - 1.530 patiënten in Nederland mogelijk in aanmerking komen voor deze behandeling. De verwachting is dat een gedeelte van de patiënten mogelijk zal overstappen van lanadelumab op berotralstat.

5. Expected cost per patient per year

There is currently nothing known about the expected cost.

6. Potential total cost per year

There is currently nothing known about the possible total cost.

7. Off label use

There is currently nothing known about off label use.

8. Indication extension

There is currently nothing known about indication extensions.

9. Other information

There is currently no futher information available.