Extended indication Orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in
Therapeutic value No estimate possible yet
Total cost 53,794,065.50
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 comments Bradykinine remmer

Registration

Registration route Centralised (EMA)
Submission date April 2020
Expected Registration April 2021
Registration phase Registered
Additional comments 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
Substantiation De verwachting is dat berotralstat (oraal) de concurrentie aan zal gaan met lanadelumab (subcutaan).
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. Het zal met name ingezet worden ter preventie. Dat maakt dat het patiëntaantal mogelijk lager gaat zijn.

Expected cost per patient per year

Cost < 153,697.33
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 comments 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

53,794,065.50

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.

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.