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Berotralstat

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.