Australian speciality biotechnology company CSL has received the European Commission (EC) conditional marketing authorisation (CMA) for Hemgenix (etranacogene dezaparvovec).

Previously known as AMT-061, Hemgenix is an adeno-associated virus (AAV)-based gene therapy that uses a specific type of AAV, dubbed AAV5, as its vector.

The gene therapy is indicated for the treatment of severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults, without a history of Factor IX inhibitors.

It was previously approved by the US Food and Drug Administration (FDA), and is currently under review by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

Hemgenix is the first and only one-time gene therapy approved in the European Union (EU) and European Economic Area (EEA), said the biotechnology company.

CSL chief medical officer and research and development head 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 hemophilia B and the healthcare professionals who treat them.

“Hemgenix, and our partnership with uniQure, underscore CSL’s promise to pursue, develop and deliver disruptive innovations when patients can benefit, particularly in disease states we know well like hemophilia B.”

According to the company, people living with haemophilia B currently require lifelong treatment of intravenous infusions of Factor IX to maintain sufficient levels.

Factor IX is a protein produced naturally in the body, which helps the blood to form clots to stop bleeding, and its frequent infusions may impact the quality of life of patients.

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) identifies an unmet medical need for new treatments that reduce frequent infusions.

The EC approval follows the CHMP’s positive opinion granted in December last year.

It was based on results from the HOPE-B trial, an ongoing, multinational, open-label, single-arm study that evaluates the safety and efficacy of Hemgenix.

In the clinical trial, patients treated with Hemgenix showed stable increases in levels of mean Factor IX activity, which led to an adjusted annualized bleed rate (ABR) reduction of 64%.

After infusion, Hemgenix led to the discontinuation of routine Factor IX prophylaxis in 96% of patients, and a 97% reduction in mean Factor IX consumption at 18 months post-treatment.

CSL Behring senior vice president and Europe commercial operations general manager Lutz Bonacker said: “At CSL Behring, our promise is simple – to save and improve lives – and we achieve this by living our core values of patient focus, innovation, integrity, collaboration, and superior performance.

“The approval of HEMGENIX in Europe is a result of that focus and a milestone for the hemophilia B community, and we now need to work to ensure that as many eligible patients across Europe can access this innovative treatment as possible.”