Novel haemophilia B gene therapy approved

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The US Food and Drug Administration (FDA) has approved the adeno-associated virus (AAV)-based gene therapy BEQVEZ™ (fidanacogene elaparvovec-dzkt) for certain adults with haemophilia B.

The treatment is indicated for adults with moderate to severe haemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening haemorrhage, Pfizer stated. It is also approved for individuals with the disease who have repeated, serious spontaneous bleeding episodes, and do not have neutralising antibodies to AAV serotype Rh74var (AAVRh74var) capsid.

About the one-time gene therapy

BEQVEZ works by introducing in the transduced cells a functional copy of the FIX gene encoding a high-activity FIX variant. This means that patients with the rare genetic bleeding disorder can produce FIX themselves instead of relying on regular intravenous infusions of FIX, which is the current standard of care, according to 2015 research highlighted by Pfizer.

Commenting on the FDA’s approval of the haemophilia B treatment, Aamir Malik, Chief US Commercial Officer and Executive Vice President, Pfizer, shared that this medicine “has the potential to offer both long-term bleed protection and value to the healthcare system because of its one-time administration.”

This authorisation of BEQVEZ is based on results from the pivotal Phase III BENEGENE-2 study.

According to clinical data, a one-time dose of the haemophilia B treatment reduced bleeds post-treatment compared to standard of care with a median of zero bleeds (range 0 to 19) after up to three years of follow-up. Pfizer stated that the gene therapy enabled sustained bleed protection, “potentially avoiding years of treatment burden with prophylaxis for many patients”.

FDA approves first gene therapy for Haemophilia B

“Many people with haemophilia 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,” explained Dr Adam Cuker, MS, Director, Penn Comprehensive and Hemophilia Thrombosis Program. “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.”

Pfizer licensed BEQVEZ from Spark Therapeutics in December 2014. BEQVEZ is currently being reviewed by the European Medicines Agency (EMA), Pfizer confirmed.