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.