FDA Approves Second Hemophilia B Gene Therapy Product

The U.S. Food and Drug Administration (FDA) has approved BEQVEZ™ (Pfizer, Inc.), a new hemophilia B gene therapy product. It is designed with bioengineered adeno-associated virus (AAVs) vectors to introduce a working copy of the factor IX gene. The therapy is administered as a one-time intravenous infusion to stimulate the long-term production of clotting factor IX (FIX) and prevent bleeding. This represents the second hemophilia B gene therapy product to be approved in the U.S. – the first was approved in the fall of 2022.

According to a Pfizer press release, BEQVEZ (fidanacogene elaparvovec-dkzt) is approved in the United States specifically for the treatment of adults with moderate to severe hemophilia B who:

The FDA approval is based on results from the phase 3 BENEGENE-2 clinical study. 

Study investigators looked at safety and efficacy in BEQVEZ-treated adult males with severe or moderate hemophilia B. A primary endpoint of the trial, and key measure of the therapy’s efficacy, is annualized bleeding rate (ABR). Investigators compared ABR of participants treated with BEQVEZ versus a standard prophylaxis regimen with a FIX replacement therapy, which was administered as part routine care during the so-called “lead-in” period lasting for at least six months.

A mean ABR of 2.5 was observed among patients who received BEQVEZ in the efficacy evaluation period – defined as between week 12 and data cutoff (median 1.8 years of follow-up). This was compared to a mean ABR of 4.5 during the lead-in pre-treatment period. 

Bleeds were eliminated in 60% of patients​​ compared to 29% in the prophylaxis arm. group. A median ABR of zero (range of 0 to 19) was observed during the efficacy evaluation period compared to the prophylaxis arm, which showed a median ABR of 1.3 (range of 0 to 53.9). 

In terms of safety, Pfizer reports that the therapy was “generally well-tolerated.” The most common adverse reaction was an increase in liver enzymes. The company also reported no deaths, serious adverse events related to treatment or associated with infusion reactions, thrombotic events, or FIX inhibitors. Visit ClinicalTrials.gov to learn more about this gene therapy trial.

Go to the FDA website to read the complete approval letter and access more product-specific information. 

“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,” said Adam Cuker, MD, 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 also announced that they are launching a BEQVEZ warranty program based on patient response to the treatment. The goal of the warranty is to offer “greater certainty” to payers and maximize access for eligible patients. It is also meant to offer financial protection by insuring against the “risk of efficacy failure.” 

“We are leveraging our expertise that comes with more than 40 years of experience in the hemophilia space, and are proactively working with treatment centers, payers, and the hemophilia community to appropriately help ensure the healthcare system is prepared to readily deliver BEQVEZ to the patients who can benefit from it,” said Aamir Malik, Chief U.S. Commercial Officer and Executive Vice President, Pfizer 

Visit the NBDF website   to learn more about treatment for bleeding disorders.

Source: Pfizer press release dated April 26, 2024

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