Berotralstat Medicine 10 June 2020 Extended indication Treatment for the Prevention of Attacks in patients with Type I and II Hereditary Angioedema Therapeutic value No judgement Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer BioCryst Portfolio holder Mechanism of action Other Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Bradykinine remmer Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date April 2020 Expected Registration May 2021 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks 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 This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat berotralstat (oraal) de concurrentie aan zal gaan met lanadelumab (subcutaan). Het zal met name ingezet worden ter preventie. Duration of treatment Frequency of administration 1 times a day Dosage per administration 150 mg References NCT03485911 Additional remarks Expected patient volume per year Patient volume < 350 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Adis Insight Additional remarks Geen nieuwe indicaties in de pijplijn Other information Additional remarks