Ravulizumab

Extended indication	Extension of indication to include treatment of paediatric patients with paroxysmal nocturnal haemoglobinuria (PNH) for Ultomiris (>10 kg).
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 remarks	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 remarks	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 remarks	PNH is op de kinderleeftijd erg zeldzaam. De diagnose wordt mogelijk bij minder dan 5 kinderen per jaar in Nederland gesteld.

Patient volume

< 5

Market share is generally not included unless otherwise stated.

References

Expertopinie

Additional remarks

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 remarks	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.

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.