CSL Behring announced that the European Medicines Agency (EMA) has granted its approval for an accelerated assessment request for etranacogene dezaparvovec Marketing Authorization Application (MAA). Etranacogene dezaparvovec is an investigational gene therapy for people with hemophilia B, a life-threatening bleeding disorder, and is currently being studied in the Phase 3 HOPE-B clinical trial.

The EMA grants accelerated assessment if a medicinal product is expected to be of major public health interest and constitutes a therapeutic innovation. This potentially reduces the assessment timeline, once the MAA is filed and validated and could speed up patient access to this gene therapy.

Etranacogene dezaparvovec has already been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to the Priority Medicine (PRIME) regulatory scheme by the EMA. CSL Behring plans to submit regulatory applications for marketing approval of etranacogene dezaparvovec in the European Union and the United States in the first half of 2022.

CSL Behring recently announced positive top line results from the HOPE-B pivotal trial of etranacogene dezaparvovec. The study achieved its primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose. It also achieved a secondary endpoint demonstrating statistical superiority in reduction of annualized bleeding rate, compared with baseline FIX prophylactic therapy. Factor IX is a naturally occurring protein produced in the body, which helps blood to clot. 

The study further showed that Etranacogene dezaparvovec was generally well-tolerated with over 80% of adverse events considered mild in the 53 patients involved in the 18-month follow-up.

"These encouraging results illustrate the potential that gene therapy has to be a long-term treatment option for patients living with hemophilia B and we look forward to sharing more detailed data with the medical community in the near future," commented Lutz Bonacker Senior Vice President and General Manager Europe at CSL Behring. "This milestone advances our efforts towards expected regulatory submissions in the first half of 2022."