The European Medicines Agency (EMA) has initiated the review of CSL Behring’s Marketing Authorization Application (MAA) for gene therapy, etranacogene dezaparvovec (EtranaDez), for haemophilia B treatment.

The agency accepted the application of the company for review under accelerated procedure.

An investigational adeno-associated virus five (AAV5)-based gene therapy, etranacogene dezaparvovec is given as a single infusion to treat haemophilia B patients who have a severe bleeding phenotype. 

On obtaining approval, etranacogene dezaparvovec will become the first-ever one-time gene therapy that can substantially lower annual bleed rates for patients in the European Union (EU) and European Economic Area. 

The application is backed by positive data from the open-label, multinational, single-arm Phase III HOPE-B clinical trial that assessed the safety and efficacy of etranacogene dezaparvovec in individuals with haemophilia B. 

Findings showed that etranacogene dezaparvovec-treated subjects with a severe bleeding phenotype had a 64% decline in their adjusted annualised bleed rate (ABR).

The treatment was found to be superior to prophylaxis treatment at 18 months, versus a six-month run-in period. 

The company also reported stable and lasting increases in mean Factor IX (FIX) activity levels, following the gene therapy administration. 

Etranacogene dezaparvovec was also reported to be well-tolerated, with mild adverse events observed.

UniQure carried out the etranacogene dezaparvovec’s multi-year clinical development until CSL Behring obtained worldwide rights to market the gene therapy. 

CSL Behring sponsored the trials in the US on gaining commercialisation rights, and is expanding it to the European Union.

CSL Behring Global Regulatory Affairs head Emmanuelle Lecomte Brisset said: “As the first gene therapy candidate for haemophilia B, this pivotal regulatory milestone brings CSL Behring one step closer to delivering on the promise of gene therapy for the bleeding disorders community. 

“We look forward to working with regulatory authorities to bring the transformative potential of gene therapy to people living with this debilitating, life-long condition.”

In May 2020, CSL Behring’s Australian subsidiary announced the development of a plasma-derived therapeutic to treat serious complications in Covid-19 patients.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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