Active Ingredient: Etranacogene dezaparvovec
Etranacogene dezaparvovec is indicated for the treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Intravenous, 20,000,000,000,000 copies etranacogene dezaparvovec per kilogram of body weight, one dose.
The recommended dose of etranacogene dezaparvovec is a single dose of 2 × 1013 gc/kg body weight, administered as an intravenous infusion.
Etranacogene dezaparvovec can be administered only once.
The onset of effect from etranacogene dezaparvovec treatment may occur within several weeks post-dose. Therefore, haemostatic support with exogenous human Factor IX may be needed during the first weeks after etranacogene dezaparvovec infusion to provide sufficient Factor IX coverage for the initial days post-treatment. Monitoring of the Factor IX activity (e.g. weekly for 3 months) is recommended post-dose to follow the patient’s response to etranacogene dezaparvovec.
When using an in vitro activated partial thromboplastin time (aPTT)-based one-stage clotting assay for determining Factor IX activity in patients' blood samples, plasma Factor IX activity results can be affected by both the type of aPTT reagent and the reference standard used in the assay. This is important to consider particularly when changing the laboratory and/or reagents used in the assay. Therefore the same assay and reagents are recommended to be used to monitor Factor IX activity over time.
In case increased plasma Factor IX activity levels are not achieved, decrease, or bleeding is not controlled or returns, post-dose testing for Factor IX inhibitors is recommended along with Factor IX activity testing.
No dose adjustments are recommended in elderly patients. Limited data are available in patients aged 65 years and older.
Etranacogene dezaparvovec is administered as a single-dose intravenous infusion.
Etranacogene dezaparvovec must not be administered as an intravenous push or bolus.
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