Pfizer will go toe-to-toe with CSL Behring following the FDA’s Friday approval of its hemophilia B gene therapy Beqvez and will launch a warranty program based on the durability of response.
Pictured: Entrance to Pfizer’s office in Belgium/iStock, Alexandros Michailidis
The FDA on Friday gave its approval to Pfizer’s one-time gene therapy fidanacogene elaparvovec-dzkt, which will now carry the brand name Beqvez, for the treatment of moderate to severe hemophilia B in adults.
Per its label, Beqvez is indicated for patients who are currently on factor IX prophylaxis or who have a history of life-threatening hemorrhage. The gene therapy can also be administered to those who have had repeated serious spontaneous bleeding episodes and are negative for neutralizing antibodies against the adeno-associated virus (AAV) serotype Rh74var.
Aamir Malik, chief U.S. commercial officer at Pfizer, in a statement said that Beqvez’s approval will provide patients with a “medicine that has the potential to offer both long-term bleed protection,” as well as add “value to the healthcare system because of its one-time administration.”
Pfizer will leverage its expertise in the hemophilia space and collaborate with treatment centers, payers and other stakeholders to “ensure the healthcare system is prepared to readily deliver Beqvez to the patients who can benefit from it,” Malike added.
As part of its efforts to ensure broad access to Beqvez, Pfizer also announced on Friday that it will launch an “innovative warranty program” based on the therapy’s durability of response, which in turn will provide “financial protection” for patients and payers by “insuring against the risk of efficacy failure.” The pharma has not yet announced a price for Beqvez.
Delivered via an AAV vector, Beqvez is a gene therapy that works by delivering a functional copy of the factor IX gene, which encodes a high-activity protein variant that restores the patient’s ability to clot blood and addresses the hallmark hemophilia B symptom of excessive bleeding. Beqvez was first approved in Canada, which handed Pfizer the regulatory win in January 2024.
Beqvez’s FDA approval on Friday was supported by data from the pivotal Phase III BENEGENE-2 study, an open-label and single-arm trial that enrolled 45 male patients with factor IX circulating activity of 2% or lower.
Results showed that a single intravenous dose of Beqvez led to a median annualized bleeding rate (ABR) of zero during the study’s efficacy evaluation period. By comparison, patients in the control arm who received a standard factor IX prophylaxis replacement regimen had a median ABR of 1.3. Beqvez also eliminated bleeding in 60% of patients versus 29% in the prophylaxis group.
In terms of safety, BENEGENE-2 found that Beqvez was generally well-tolerated with no serious adverse events or deaths associated with the gene therapy or with infusion reactions. There were also no thrombotic events reported in the study. Pfizer will continue to monitor patients for 15 years for long-term durability and safety.
With Beqvez’s approval, Pfizer now follows in the footsteps of CSL Behring, which secured the FDA’s greenlight for its own hemophilia gene therapy Hemgenix (etranacogene dezaparvovec-drlb) in November 2022. For the full year ended June 2023, Hemgenix contributed to the nearly nearly $1.2 billion revenue of the company’s Hemophilia business.
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
