An adeno-associated virus vector-based gene therapy, Hemgenix, was approved for adults with haemophilia B by the US Food and Drug Administration (FDA) in November 2022 (see BioNews 1169).
Now, the European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation in the European Union for the drug.
The EMA's recommendation is based on the results of two studies, which evaluated the safety and effectiveness of Hemegenix in 57 men aged between 18 and 75 years with severe or moderately severe haemophilia B.
In the first study involving three patients, all had sustained positive treatment effects up to three years following the infusion. In the second study, 52 patients sustained positive treatment effects up to two years following the infusion.
Read more on the EMA's recommendation here.
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