First gene therapy for haemophilia B gets go ahead in Europe

Gene therapy for haemophilia

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). 

The CHMP has recommended conditional marketing authorisation of CSL’s etranacogene dezaparvovec, a one-time gene therapy for the treatment of appropriate adults with the disease. 

The therapy was also recently approved by the FDA for patients in the US. 

Emmanuelle Lecomte Brisset, Senior Vice President and Head of Global Regulatory Affairs at CSL, said: “Getting a new medicine to this stage of the regulatory process takes the support of many, including clinical trial participants, the haemophilia 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.”   

The positive opinion is based on findings from the pivotal HOPE-B trial, which showed that patients treated with etranacogene dezaparvovec demonstrated durable increases in mean Factor IX activity levels which led to an adjusted annualised bleed rate (ABR) reduction of 64%. 

Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis. 

The clinical development of etranacogene dezaparvovec was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment. 

World’s most expensive drug

A one-time treatment with etranacogene dezaparvovec costs US$3.5 million, making it the world’s most expensive drug. 

Concerns about the high price tag have been raised in an article in Nature, particularly whether it will be affordable for low to middle-income countries. 

CSL has responded in a statement that the treatment actually represents a saving on the current costs of patients receiving routine FIX prophylaxis once or twice weekly. 

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