Extended indication Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemog
Therapeutic value No estimate possible yet
Total cost 1,400,000.00
Registration phase Positive CHMP opinion

Product

Active substance Ravulizumab
Domain Cardiovascular diseases
Reason of inclusion Indication extension
Main indication Other non-oncological hematological medications
Extended indication Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH) for Ultomiris (>10 kg).
Proprietary name Ultomiris
Manufacturer Alexion
Mechanism of action Complement inhibitor
Route of administration Intravenous
Therapeutical formulation Intravenous drip
Budgetting framework Intermural (MSZ)
Additional comments Monoclonaal antilichaam gericht tegen het C5 complement eiwit.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date January 2021
Expected Registration December 2021
Orphan drug Yes
Registration phase Positive CHMP opinion
Additional comments Positieve CHMP-opinie in juli 2021. Ravulizumab is breed uitgesloten, deze indicatie zal dus ook in de sluis worden geplaatst.

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

Patient volume

< 5

Market share is generally not included unless otherwise stated.

References Expertopinie
Additional comments PNH is op de kinderleeftijd erg zeldzaam. De diagnose wordt mogelijk bij minder dan 5 kinderen per jaar in Nederland gesteld.

Expected cost per patient per year

Cost < 280,000.00
Additional comments Voor volwassenen is behandeling met ravulizumab €280.000 in het eerste jaar en ongeveer €260.000 in de daar opvolgende jaren. De kosten zullen bij pediatrische patiënten mogelijk lager uitvallen.

Potential total cost per year

Total cost

1,400,000.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.