FDA Approves Pfizer’s First Gene Therapy for Haemophilia B

The United States Food and Drug Administration (FDA) has, on Friday, granted approval to Pfizer for its first-ever gene therapy. This marks a significant milestone in the treatment of a rare genetic bleeding disorder called Haemophilia B.

Individuals affected by hemophilia have a genetic anomaly in a specific gene responsible for controlling the production of clotting factor proteins. This condition can lead to both mild and severe bleeding episodes, particularly after injury or surgical procedures.

Pfizer’s gene therapy, marketed as Beqvez, targets adults with moderate to severe hemophilia B. This option offers a new avenue for managing this debilitating condition.

According to a spokesperson from Pfizer, Beqvez will be available by prescription to eligible patients in the current quarter. However, the therapy comes with a substantial price tag of $3.5 million before insurance and rebates, making it one of the most expensive drugs in the U.S. healthcare landscape.

Hemophilia B affects over 7,000 individuals in the U.S., predominantly males, and is characterised by insufficient levels of factor IX, a protein crucial for blood clotting. Beqvez is a one-time treatment and aims to address this deficiency by enabling patients to produce factor IX themselves, thereby preventing and controlling bleeding episodes.

Clinical trials have demonstrated Beqvez’s superiority over traditional treatment methods, which often involve administering factor IX infusions multiple times a week or month. The therapy offers potential long-term benefits, reducing both the medical and treatment burden associated with hemophilia B.

“For people living with hemophilia, disease management can interfere with many aspects of their lives. A one-time infusion of BEQVEZ may allow eligible patients more time for the things they love

“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues.

“A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term”, said Adam Cuker, M.D., M.S., Director, Penn Comprehensive and Hemophilia Thrombosis Program.

Beqvez joins the ranks of other high-cost gene therapies, such as Hemgenix from CSL Behring, approved for hemophilia B treatment. Despite the promise of gene therapies, challenges such as cost, logistical issues, and patient satisfaction with current treatments have tempered their widespread adoption.

Pfizer is also invested in other gene and cell therapies, including treatments for other genetic disorders such as Duchenne muscular dystrophy.

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