Berotralstat Medicine 10 June 2020 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. Therapeutic value No estimate possible yet Total cost € 53,793,950.00 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. Current proprietary name Already available biosimilars / generics Proprietary name Orladeyo 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 April 2021 Orphan drug Unknown Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in februari 2021. Therapeutic value Current treatment options C1-esteraseremmers Firazyr en Cinryze. Daarnaast ook de monoklonale antistof lanadelumab. Therapeutic value No estimate possible yet 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). 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. Het zal met name ingezet worden ter preventie. Dat maakt dat het patiëntaantal mogelijk lager gaat zijn. Expected cost per patient per year Cost € < 153,697.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References * Available list prices on 18 July 2022: PPP France €11.954,44 (= FAP of €11.921,94 (1) plus distributor margin(2) of €32,50); PPP UK £10.205 (3) (= €12.047,27); PPP Norway NOK116.600 (4) (= €11.369,73), exchange rates as of 18 July 2022 (5); PPP: Pharmacy Purchasing Price 1.https://www.legifrance.gouv.fr/loda/id/JORFTEXT000000843609/2021-03-01 2.https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000045960460?init=true&page=1&query=berotralstat&searchField=ALL&tab_selection=all 3.https://www.nice.org.uk/guidance/ta738/documents/129 4. https://legemiddelverket.no/english/public-funding-and-pricing/maximum-price#list-of-products-with-maximum-prices 5.https://www.ecb.europa.eu/stats/policy_and_exchange_rates/euro_reference_exchange_rates/html/index.en.html Additional remarks The average list price for one pack of Orladeyo® containing 28 capsules in the relevant price reference countries is €11.790,48*. The annual per patient cost for treatment with Orladeyo® is €153.697,33. Potential total cost per year Total cost € 53,793,950.00 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