NEW YORK – Pfizer has discontinued its gene therapy for hemophilia B, Beqvez (fidanacogene elaparvovec), for which the company received regulatory approvals in the US and elsewhere last year.
Pfizer in a statement to Precision Medicine Online said Beqvez, which carried a wholesale acquisition cost of $3.5 million in the US, had seen limited interest from physicians and patients.
"Pfizer has made the decision to cease further development and commercialization activities with respect to Beqvez for several reasons, including the limited interest patients and their doctors have demonstrated in hemophilia gene therapies to date," a Pfizer spokesperson wrote in an emailed statement.
Pfizer's Beqvez, which was licensed from Spark Therapeutics in 2014, was the second gene therapy for hemophilia B to reach the market, following the US Food and Drug Administration's approval of CSL Behring's Hemgenix (etranacogene dezaparvovec) in 2022.
Beqvez was approved last year in the US, Canada, and Europe for patients with moderate to severe forms of hemophilia B based on data from an open-label Phase III trial, in which investigators found a single dose of Beqvez was better at reducing bleeds than the typical treatment for hemophilia B, routine prophylactic infusions of factor IX replacement therapy.
Patients with hemophilia B, a rare and hereditary bleeding disorder, have certain mutations in the F9 gene that result in lack of factor IX, a protein that helps blood to clot. Beqvez is designed to treat that problem by delivering an F9 transgene that encodes a high-activity variant of factor IX, so that patients can produce the protein themselves.
In December, Pfizer also terminated a collaboration and license agreement with Sangamo Therapeutics, returning development and commercialization rights to Sangamo for the hemophilia A gene therapy giroctocogene fitelparvovec, despite Pfizer saying in July that it planned to discuss positive top-line Phase III trial data with regulators.
The pharmaceutical giant said, moving forward, it will focus its efforts and "rededicate" time and resources to treatments it's betting will have the greatest impact on patients, such as Hympavzi (marstacimab), an anti-tissue factor pathway inhibitor and another drug for hemophilia A and B for which Pfizer received regulatory approvals in the US and EU last year.