Extended indication Sickle cell disease; treatment of vaso-occlusive crisis.
Therapeutic value No judgement
Registration phase Clinical trials

Product

Active substance Rivipansel Sodium
Domain Cardiovascular diseases
Main indication Other non-oncological hematological medications
Extended indication Sickle cell disease; treatment of vaso-occlusive crisis.
Manufacturer Pfizer
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)
Particularity New medicine
Submission date 2019
Expected Registration 2020
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Current treatment options hydroxycarbamide
Therapeutic value No judgement

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 1000 patiënten met SCZ(bron:hematologienederland). 90% van alle SCZ patiënten komt mogelijk in aanmerking voor deze behandeling (bron SPS). Gezien er nog een middel 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 1750 euro voor hydroxycarbamide (GIP-databank).

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.