Extended indication

Treatment for the Prevention of Attacks in patients with Type I and II Hereditary Angioedema

Therapeutic value

No judgement

Registration phase

Registration application pending

Product

Active substance

Berotralstat

Domain

Chronic immune diseases

Reason of inclusion

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

Manufacturer

BioCryst

Mechanism of action

Other

Route of administration

Oral

Therapeutical formulation

Capsule

Budgetting framework

Extramural (GVS)

Additional comments
Bradykinine remmer

Registration

Registration route

Centralised (EMA)

Submission date

April 2020

Expected Registration

May 2021

Orphan drug

Yes

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.

Therapeutic value

Current treatment options

C1-esteraseremmers en icatibant en Cinryze. Daarnaast ook de monoklonale antistof lanadelumab.

Therapeutic value

No judgement

Substantiation

De verwachting is dat berotralstat (oraal) de concurrentie aan zal gaan met lanadelumab (subcutaan). Het zal met name ingezet worden ter preventie.

Frequency of administration

1 times a day

Dosage per administration

150 mg

References
NCT03485911

Expected patient volume per year

Patient volume

< 350

Market share is generally not included unless otherwise stated.

References
Cicardi M, Bork K, Caballero T, Craig T, Li HH, Longhurst H, Reshef A, Zuraw B, HAWK (Hereditary Angioedema International Working Group).
Allergy. 2012 Feb; 67(2):147-57.
Additional comments
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. De verwachting is dat een gedeelte van de patiënten mogelijk zal overstappen van lanadelumab op berotralstat.

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension

No

References
Adis Insight
Additional comments
Geen nieuwe indicaties in de pijplijn

Other information

There is currently no futher information available.