Pfizer wins approval for $3.5m hemophilia B gene therapy

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

The gene therapy is the third such product approved by the US Food and Drug Administration (FDA), following the November 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec) – also for hemophilia B – and the July 2023 approval of BioMarin’s Roctavian (valoctocogene roxaparvovec) for hemophilia A.

While there are two one-time therapies now available, the standard of care for hemophilia B treatment continues to be prophylactic infusions of factor XI replacement therapy, which temporarily replaces or supplements low levels of blood-clotting factor.

Pfizer has set a list price of $3.5 million for Beqvez, matching the wholesale acquisition cost of Hemgenix.

Pfizer licensed the Beqvez from gene therapy pioneer Spark Therapeutics (now part of Roche) in December 2014. The therapy forms part of the Big Pharma’s gene therapy ambitions, which has been built out through the acquisition of Bamboo Therapeutics in 2016 and a collaboration with Sangamo Therapeutics in 2020. The former added a candidate for Duchenne Muscular Dystrophy (DMD), while the latter focuses on hemophilia A.

The firm has also bulked out its manufacturing network to support gene therapies through an $800 million (originally $500 million) investment, which included the construction of an 85,500 square-foot plant in Durham, North Carolina to support clinical manufacturing.