Pfizer Secures Health Canada Approval for Beqvez, Its First Gene Therapy for Treating Hemophilia B

Health Canada’s Nod to Pfizer’s Gene Therapy

Pfizer starts 2024 with the approval of its gene therapy, Beqvez (fidanacogene elaparvovec), by Health Canada. This approval is for adults aged 18 and older with a moderately-severe to severe form of hemophilia B, who do not have neutralizing antibodies against AAV serotype Rh74.

Beqvez: How It Works

introduces a high-activity human FIX gene to enable patients to produce the FIX clotting protein endogenously, a departure from the regular intravenous infusions. The Phase III BENEGENE-2 study, crucial for this approval, showed that Beqvez effectively reduced annualized bleeding rates compared to standard FIX prophylaxis.

Hemophilia B: A Brief Overview

Hemophilia B is a genetic disorder characterized by insufficient FIX clotting protein, leading to prolonged bleeding, especially into joints and muscles. It affected over 38,000 people globally in 2021, with more than 700 cases in Canada. The disorder is typically X-linked recessive and primarily affects males.

FDA Review and Pfizer’s Strategic Shift

While Health Canada has granted approval, Pfizer awaits a decision from the US Food and Drug Administration (FDA), expected by mid-2024. Concurrently, Pfizer has announced a shift in its strategy, moving away from developing viral vector-based gene therapies.

Financial Repercussions and Strategy Adjustment

Facing reduced sales from its COVID-19 vaccine and antiviral products, Pfizer is implementing a $4 billion cost-cutting initiative, heavily affecting research and development. Part of its strategic realignment involves a potential $1 billion deal transferring some of its assets and programs to Alexion, a division of AstraZeneca.

Market Dynamics: Beqvez and Hemgenix

With the approval of Beqvez, Pfizer enters a market that includes CSL Behring’s Hemgenix (etranacogene dezaparvovec), another gene therapy for hemophilia B. Hemgenix, priced at $3.5 million, was approved by the FDA in November 2022 and Health Canada in October 2023. The price of Beqvez is yet to be announced.

Conclusion: The Entry of Beqvez into Genetic Medicine

The approval of Beqvez by Health Canada introduces a new gene therapy option for hemophilia B. As Pfizer adjusts its business strategy, the healthcare community awaits the potential impact of Beqvez on genetic medicine treatments.

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