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Berotralstat

Orladeyo

Extended indication Orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in
Therapeutic value No estimate possible yet
Registration phase Registered

Product

Active substance Berotralstat
Domain Chronic immune diseases
Reason of inclusion New medicine (specialité)
Main indication Other chronic immune diseases
Extended indication Orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.
Proprietary name Orladeyo
Manufacturer BioCryst
Mechanism of action Other
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)
Additional remarks Bradykinine remmer

Registration

Registration route Centralised (EMA)
Submission date April 2020
Expected Registration April 2021
Orphan drug Yes
Registration phase Registered
Additional remarks Datum indiening en verwachte registratie op basis van actual primary completion date van fase 3 studie in April 2019. Positieve CHMP-opinie in februari 2021.

Therapeutic value

Current treatment options C1-esteraseremmers en icatibant en Cinryze. Daarnaast ook de monoklonale antistof lanadelumab.
Therapeutic value No estimate possible yet
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 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. 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 remarks Geen nieuwe indicaties in de pijplijn

Other information

There is currently no futher information available.