Recently, Pfizer announced that Health Canada has approved BEQVEZ (fidanacogene elaparvovec) for marketing. This is an AAV vector-based gene therapy used to treat moderate to severe hemophilia B patients who test negative for neutralizing antibodies against AAV serotype Rh74.Adult(18 years old and above)Patient。
BEQVEZ is an in vivo gene therapy drug that uses genetically engineered AAV.The capsid enhances the ability to deliver the highly active human FIX gene to target tissues and organs in the body, with administration via intravenous infusion, providing long-term or even lifelong efficacy with a single dose.Health Canada approved BEQVEZ based on an open-label, single-arm Phase III BENEGENE-2 study, which aimed to evaluate the efficacy and safety of fidanacogene elaparvovec in male participants aged 18 to 65 with moderate to severe hemophilia B. This study enrolled a total of 45 participants.Clinical data shows,BENEGENE-2 TrialThe primary endpoint was met.Compared with traditional conventional treatmentPlan, AcceptBEQVEZPatients receiving infusions demonstrated good efficacy and safety in terms of the annualized bleeding rate (ABR).，Pfizer's previously released data showed that this gene therapy successfully reduced patients' ABR by 71%.The annualized coagulation factor infusion rate decreased by 92%. In addition,AcceptBEQVEZAfter infusion, the FIX activity in the patient remained stable.
Wendy Quinn, President of the Canadian Hemophilia Society (CHS), stated:"The Canadian Hemophilia Society (CHS) welcomes the approval of BEQVEZ for marketing, and we hope that eligible patients will have access to gene therapy. Gene therapy has the potential to fundamentally transform treatment approaches for certain individuals and improve their quality of life. The CHS will work closely with health authorities across Canada to ensure that gene therapy for hemophilia B becomes available nationwide to eligible patients as soon as possible."Source of reference materials: Pfizer's official website, the WeChat Official Account "Cell and Gene Therapy Field," etc.
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