Extended indication In paediactric and adult patients with Recurrent Pericarditis ≥12 years
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Rilonacept
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other medication for cardiovascular diseases
Extended indication In paediactric and adult patients with Recurrent Pericarditis ≥12 years
Manufacturer Regeneron
Route of administration Subcutaneous
Therapeutical formulation Injection
Budgetting framework Extramural (GVS)
Additional comments Werkingsmechanisme: "Recombinant fusion protein that blocks IL-1α and IL-1β signaling"

Registration

Registration route Centralised (EMA)
ATMP No
Submission date 2021
Expected Registration 2022
Orphan drug No
Registration phase Clinical trials
Additional comments Indieningsdatum op basis van primary completion datum fase 3 studie in mei 2020.

Therapeutic value

Current treatment options Anti-inflammatoire geneesmiddelen.
Therapeutic value No estimate possible yet
Substantiation In de RHAPSODY fase 3 studie placebo-gecontroleerd, had 7% in de rilonacept groep een pericarditis recurrence, vergeleken met 74% in de placebo groep. Mediane tijd tot recurrence: hazard ratio, 0.04; 95% CI, 0.01 to 0.18; P<0.001. Een interleukine remmer zal niet de eerste keuze zijn in het geval van een recurrent pericarditis. De verwachting is dat colchicine eerder in de behandelrichtlijn gegeven zal worden.
Frequency of administration 1 times a week
Dosage per administration 320 mg
References NCT03737110

Expected patient volume per year

Patient volume

< 30

Market share is generally not included unless otherwise stated.

References http://echocardiografie.nl/wp-content/uploads/2019/06/Pericarditis-2019.pdf
Additional comments Diagnose Pericarditis wordt zo'n 28 keer per 100.000 personen per jaar gesteld. Het aantal recidief pericarditiden wordt geschat op circa 300 per jaar. Een klein percentage hiervan zal mogelijk met dit geneesmiddel behandeld kunnen gaan worden. Dit zal maximaal 10% zijn. Hierdoor komen er maximaal 30 patiënten per jaar in aanmerking.

Expected cost per patient per year

References https://www.pharmaceuticalcommerce.com/view/the-worlds-most-expensive-drug-alexion-pharmaceuticals-soliris-at-409500-per-year
Additional comments De jaarlijkse kosten in de VS worden geschat op 250.000 dollar maximaal per patiënt.

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 Yes
Indication extensions Inborn genetic disorders, Urticaria
References Adis Insight

Other information

There is currently no futher information available.