Biotechnology company CSL has received conditional marketing authorisation (CMA) from the European Commission (EC) for its Hemgenix (etranacogene dezaparvovec) to treat adult patients with haemophilia B (congenital Factor IX deficiency).
The adeno-associated virus five (AAV5)-based gene therapy, Hemgenix, is intended to treat adult patients with moderately severe and severe haemophilia B without a Factor IX inhibitors history.
It uses a specific type of AAV as its vector, AAV5, which carries the naturally occurring Padua gene variant of Factor IX (Factor IX-Padua) that generates Factor IX proteins.
Hemgenix is claimed to be the first gene therapy to receive approval for treating haemophilia B in the EEA and the EU.
CSL chief medical officer and Research & Development head Dr Bill Mezzanotte said: “The approval of Hemgenix in Europe is the essence of great science delivering a medicine that we believe can transform the treatment paradigm for both people living with haemophilia B and the healthcare professionals who treat them.”
The regulatory approval followed a positive opinion from Committee for Medicinal Products for Human Use (CHMP) in December last year.
Its decision is based on the data obtained from the ongoing, open-label, multinational, single-arm, pivotal Phase III HOPE-B trial.
The trial is designed to evaluate the safety and efficacy of Hemgenix.
According to the findings, haemophilia B patients treated with Hemgenix had stable and durable increases in mean Factor IX activity levels.
This led to a 64% reduction in an adjusted annualised bleed rate (ABR).
Additionally, 96% of subjects who received Hemgenix stopped Factor IX prophylaxis usage and had a 97% decline in mean Factor IX consumption at 18 months after treatment.
In December last year, CSL subsidiary CSL Seqirus finalised a global collaboration and licence agreement with Arcturus Therapeutics.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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