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Efanesoctocog alfa

Extended indication

Treatment and prophylaxis of bleeding in patients with haemophilia A
Therapeutic value

No estimate possible yet
Registration phase

Registration application pending

Product

Active substance

Efanesoctocog alfa
Domain

Cardiovascular diseases
Reason of inclusion

New medicine (specialité)
Main indication

Other non-oncological hematological medications
Extended indication

Treatment and prophylaxis of bleeding in patients with haemophilia A
Manufacturer

Sobi
Portfolio holder

Sobi
Mechanism of action

Coagulant
Route of administration

Intravenous
Therapeutical formulation

Intravenous drip
Budgetting framework

Intermural (MSZ)
Additional remarks 
Factor VIII replacement.

Registration

Registration route

Centralised (EMA)
Type of trajectory

Normal trajectory
ATMP

No
Submission date

May 2023
Expected Registration

June 2024
Orphan drug

Yes
Registration phase

Registration application pending

Therapeutic value

Therapeutic value

No estimate possible yet
Frequency of administration

1 times a week
Dosage per administration

50 UI/kg
References 
NCT05817812 (Freedom)

Expected patient volume per year

There is currently nothing known about the expected patient volume.

Expected cost per patient per year

Cost

< 260,000.00
References 
https://www.biopharmadive.com/news/sanofi-bioverativ-fda-approve-drug-rare-anemia/618416/
Additional remarks 
In de Verenigde Staten is de lijstprijs bekend. Dit is 280.000 dollar per patiënt per jaar voor een gemiddelde patiënt van 75 kg (1800 dollar per dosis). Omgerekend naar euro is dat €260.000 . De prijs in Nederland is nog niet bekend. Waarschijnlijk zal deze prijs lager zijn in Nederland.

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.