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Rivipansel sodium

Extended indication Treatment of Vaso-Occlusive Crisis in patients With Sickle Cell Disease
Therapeutic value No judgement yet
Registration phase Clinical trials

Product

Active substance Rivipansel sodium
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Treatment of Vaso-Occlusive Crisis in patients With Sickle Cell Disease
Manufacturer Pfizer
Mechanism of action Other
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional remarks Small molecule glycomimetic inhibitor of E-, P- and L-selectins.

Registration

Registration route Centralised (EMA)
Submission date 2021
Expected Registration 2022
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Fabrikant verwacht registratie in H1 2022

Therapeutic value

Current treatment options hydroxycarbamide
Therapeutic value No judgement yet
Substantiation In de enige fase 2 studie (Telen et al, Blood 2015) was er geen verschil in de primaire uitkomstmaat tussen rivipansel en placebo. Een fase 3 studie is ongoing
Duration of treatment Average 3.5 day / days
Frequency of administration 2 times a day
Additional remarks oplaaddosis 20 mg/kg, gevolgd door elke 12 uur 10 mg/kg tot verbetering van de crise en met een maximum van 15 giften.

Expected patient volume per year

Patient volume

< 450

Market share is generally not included unless otherwise stated.
References http://www.hematologienederland.nl, https://www.sps.nhs.uk
Additional remarks In Nederland leven naar schatting 1.000 patiënten met SCZ. Ongeveer 90% van alle SCZ patiënten komt mogelijk in aanmerking voor deze behandeling. Gezien er nog een geneesmiddel ontwikkeld wordt voor dezelfde indicatie is de inschatting dat maximaal 450 patiënten voor deze behandeling in aanmerking komen.

Expected cost per patient per year

References GIP-databank
Additional remarks Vergoeding per gebruiker in 2015 betrof zo'n €1.750 voor hydroxycarbamide.

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 No

Other information

There is currently no futher information available.