CSL closer to gene therapy treatments for hemophilia B patients in Europe

haemophilia hemophilia

CSL says the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. 

Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion. 

If the marketing authorization for which CSL applied under the brand name HEMGENIX is approved by the European Commission, etranacogene dezaparvovec would be the first gene therapy for people living with hemophilia B in the European Union (EU) and European Economic Area (EEA). 

“The CHMP’s positive opinion moves us one step closer to bringing this ground-breaking innovation to hemophilia B patients in Europe,” said Emmanuelle Lecomte Brisset, senior vice president and head of global regulatory affairs at CSL. 

“Getting a new medicine to this stage of the regulatory process takes the support of many, including clinical trial participants, the hemophilia community in general, investigators, clinicians, regulatory agencies, our people, and our partners at uniQure to name a few. Thank you to all for the role you have played in helping us reach this regulatory milestone in Europe.”  

CSL’s gene therapy positive opinion based on pivotal trial

The positive CHMP opinion is based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. 

These findings showed that hemophilia B patients treated with etranacogene dezaparvovec demonstrated stable and durable increases in mean Factor IX (FIX) activity levels (with a mean FIX activity of 36.9%) which led to an adjusted annualized bleed rate (ABR) reduction of 64%. Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.

The HOPE-B study 24 months analysis showed a sustained and durable effect of etranacogene dezaparvovec. In a clinical setting, etranacogene dezaparvovec continued to be generally well-tolerated with no serious treatment-related adverse events.

The positive opinion from the CHMP will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union member states. 

The multi-year clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment. Etranacogene dezaparvovec was recently approved by the U.S. Food and Drug Administration.

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