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Pozelimab

Extended indication Treatment of patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab therapy
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Pozelimab
Domain Cardiovascular diseases
Reason of inclusion New medicine (specialité)
Main indication Other non-oncological hematological medications
Extended indication Treatment of patients with paroxysmal nocturnal hemoglobinuria who switch from eculizumab therapy
Manufacturer Regeneron
Route of administration Intravenous
Budgetting framework Intermural (MSZ)
Additional remarks Complement C5 inhibitors

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date 2025
Expected Registration 2026
Orphan drug No
Registration phase Clinical trials
Additional remarks NCT05131204

Therapeutic value

Therapeutic value No estimate possible yet
Substantiation Er lopen nog diverse studies waarbij pozelimab gecombineerd wordt met eem siRNA cemdisiran.

Expected patient volume per year

Patient volume

< 80

Market share is generally not included unless otherwise stated.
References Expertopinie (1)
Additional remarks Op basis van de gebruikersaantallen van eculizumab bij PNH patiënten wordt er verwacht dat er maximaal 80 patiënten in aanmerking komen indien er substitutie plaatsvindt.

Expected cost per patient per year

References drugs.com
Additional remarks De Amerikaanse prijs voor pozelimab is (bbfg 200mg/ml) $36,460 voor 2ml. De prijs in de Verenigde Staten ligt vaak hoger dan de Nederlandse prijs.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.