Home Pfizer's One-Time Gene Therapy Beqvez Approved for Hemophilia B, Significantly Reducing Bleeding Episodes

Pfizer's One-Time Gene Therapy Beqvez Approved for Hemophilia B, Significantly Reducing Bleeding Episodes

Jan 05, 2024 08:43 CST Updated 07:38
Pfizer

Pharmaceutical R&D Developer

▎WuXi

Edited by Kant Content Team

Recently, Pfizer announced that Health Canada has approved its gene therapy Beqvez (fidanacogene elaparvovec) for marketing. It is used to treat adult patients aged 18 years or older with moderate to severe or severe hemophilia B who do not have neutralizing antibodies targeting the AAV serotype Rh74 subtype.The US and European regulatory authorities have accepted the listing application for Beqvez, with the PDUFA date set for the second quarter of 2024.

Hemophilia B is a life-threatening degenerative disease, in which patients lack clotting factor IX due to genetic mutations.FIX)Patients with this disease are prone to bleeding in the joints, muscles, and internal organs, resulting in pain, swelling, and joint damage. Current treatments include lifelong prophylactic infusions of coagulation factor IX to temporarily replace or supplement the low levels of the clotting factor.

▲ In

In the BENEGENE-2 trial,

Beqvez Reduces Patients' Annualized Bleeding Rate (Image Source: Reference [3])

Health Canada's approval of Beqvez is primarily based on the single-arm, open-label Phase 3 clinical trial BENEGENE-2. This study aims to evaluate the efficacy and safety of Beqvez in treating adult male subjects (aged 18-65) with moderate to severe or severe Hemophilia B (defined as ≤2% circulating FIX activity). A total of 45 patients were enrolled in this trial. The analysis showed that the BENEGENE-2 trial met its primary endpoint, demonstrating non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeding events after Beqvez infusion compared to prophylactic FIX treatment.According to the positive data from BENEGENE-2 released by Pfizer in December 2022, the gene therapy reduced patients' ABR by 71% and the annualized infusion rate of clotting factors by 92%.

▲ In

In the BENEGENE-2 trial, patients received

After Beqvez infusion, FIX activity remained stable in the body (Image source: Reference [3]).

The treatment was generally safe, with no deaths or thrombotic events reported. BENEGENE-2 recorded 14 serious adverse events, two of which were considered related to gene therapy.

Beqvez is a novel gene therapy under research, containing bioengineered AAV capsids and

High-activity variant of the FIX gene.

For patients with hemophilia B, the goal of this gene therapy is to enable them to produce their own FIX protein through a one-time treatment, rather than requiring regular intravenous infusions of FIX as is currently the standard treatment. In December 2014, Pfizer acquired Beqvez from Spark Therapeutics for a $20 million upfront payment.

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