Expand all sections

Rilzabrutinib

Extended indication Voor de behandeling van trombocytopenie bij volwassenen en adolescenten met persistente of chronisch
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Rilzabrutinib
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Voor de behandeling van trombocytopenie bij volwassenen en adolescenten met persistente of chronische immuun gemedieerde trombocytopenische purpura (ITP)
Manufacturer Sanofi
Portfolio holder Sanofi
Mechanism of action Tyrosine kinase inhibitor
Route of administration Oral
Therapeutical formulation Film-coated tablet
Budgetting framework Extramural (GVS)
Centre of expertise UMC Utrecht, LUMC, Hagaziekenhuis, UMCG

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date October 2024
Expected Registration November 2025
Orphan drug Yes
Registration phase Registration application pending
Additional remarks Fabrikant verwacht registratie het vierde kwartaal van 2025.

Therapeutic value

Current treatment options Human normal immunoglobulin, Nplate (romiplostim), Revolade (eltrombopag), Tavlesse (fostamatinib) and certain corticosteroids. Some patients required surgery (splenectomy) to remove the spleen
Therapeutic value No estimate possible yet
Substantiation Nog geen fase 3-studieresultaten bekend.
Duration of treatment continuous
Frequency of administration 2 times a day
Dosage per administration 400 mg
References Kuter, et al. N Engl J Med. 2022 (1); Kuter et al. Blood Adv 2024 (2); NCT04562766 (LUNA 3).

Expected patient volume per year

Patient volume

32 - 75

Market share is generally not included unless otherwise stated.
References ITP Expertisenetwerk (1); Kuter et al. Blood Adv. 2024 (2); Ministerie van VWS. Kerncijfers bevolking (3); Trinity RWE Claims Analysis (2020) using IBM MarketScan and CMS FFS 5% sample from 2016-2018, projected to US population using 2018 US census data (https://www.census.gov/); n (primary + secondary ITP) = 8,544 (4); Trinity RWE Line of Therapy Analysis (2020) using IBM MarketScan Commercial claims (for patients <65) and Medicare Supplemental data (for patients >=65) from 2017-2018; n = 1,716 (5).
Additional remarks Er zijn 1.450-3.350 prevalente patiënten in Nederland (2). In de Nederlandse praktijk zullen patiënten refractair op immunomodulerende medicatie en na refractair te zijn op minimaal 1 TPO-RA in aanmerking komen voor behandeling met rilzabrutinib. Amerikaanse Trinity Health data laten zien dat van de ITP populatie 25% hiervoor wordt behandeld, waarvan 9% vervolgens refractair is op TPO-RAs en in aanmerking zouden komen voor behandeling met rilzabrutinib. Toepassing van de percentages op de Nederlandse populatie komt uit op 32 tot 75 patiënten (4,5).

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

Off label use No

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.