Rivipansel sodium Medicine 12 June 2018 Extended indication Treatment of Vaso-Occlusive Crisis in patients With Sickle Cell Disease Therapeutic value No judgement yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Pfizer Portfolio holder Mechanism of action Other Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Small molecule glycomimetic inhibitor of E-, P- and L-selectins. Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date 2021 Expected Registration 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Fabrikant verwacht registratie in H1 2022 Therapeutic value Current treatment options hydroxycarbamide Therapeutic value No judgement yet This assessment does not indicate any potential inclusion in the package. 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 Dosage per administration References 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. Maximum patient volume for sluice 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 Cost References GIP-databank Additional remarks Vergoeding per gebruiker in 2015 betrof zo'n €1.750 voor hydroxycarbamide. Potential total cost per year Total cost 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 Additional remarks Other information Additional remarks