EC approves first gene therapy for haemophilia B

A conditional marketing authorisation (CMA) has been granted by the European Commission (EC) for HEMGENIX^® (etranacogene dezaparvovec), the first and only one-time gene therapy for haemophilia B (congenital Factor IX deficiency).

The treatment is indicated for severe and moderately severe forms of the condition in adults without a history of Factor IX inhibitors. HEMGENIX is the first approved gene therapy for haemophilia B in the European Union (EU) and European Economic Area (EEA).

Granting a conditional marketing authorisation for HEMGENIX

The EC’s decision follows the CHMP’s positive opinion in December 2022, based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in haemophilia B to date.

According to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), there is an unmet medical need for new therapeutic approaches that might free patients from the burden of frequent infusions, or episodically at the time of a bleeding event.

The HOPE-B trial of the first gene therapy for haemophilia B

In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds with a single infusion by delivering a functional gene that acts as a blueprint for coagulation Factor IX. The research observed that 96 percent of patients discontinued routine Factor IX prophylaxis. Mean Factor IX consumption was reduced by 97 percent at 18 months post-treatment, compared to the lead-in period.

The data showed that haemophilia B patients treated with HEMGENIX demonstrated stable and durable increases in mean Factor IX activity levels (with a mean Factor IX activity of 36.9 percent).

In a clinical setting, the treatment is generally well-tolerated with no serious treatment-related adverse events (AEs), according to data from the HOPE-B trial.

“Data from the HOPE-B study demonstrate the potential of HEMGENIX to remove the need for routine prophylaxis, by providing durable Factor IX activity, as well as improved bleeding outcomes and quality of life,” explained Professor Wolfgang Miesbach, Head of Coagulation Disorders at the Comprehensive Care Center, University Hospital of Frankfurt.

“This approval marks an important step forward in the treatment of haemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of Factor IX products,” added Professor Miesbach.

The US Food and Drug Administration (FDA) approved HEMGENIX in November 2022.