FDA approves one-time gene therapy Beqvez for hemophilia B
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The FDA approved fidanacogene elaparvovec-dzkt for treatment of adults with moderate to severe hemophilia B.
The indication applies to patients who use factor IX prophylaxis therapy, or those who have current or historical life-threatening hemorrhage, or those who have had repeated serious, spontaneous bleeding episodes and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var capsid as detected by an FDA-approved test.
Fidanacogene elaparvovec-dzkt (Beqvez, Pfizer) is an adeno-associated virus-based gene therapy. The therapy is designed to introduce in transduced cells a functional copy of the factor IX gene encoding a high-activity factor IX variant, according to a Pfizer press release.
The therapy is intended to help patients with hemophilia B produce factor IX, eliminating the need for frequent infusions that temporarily replace or supplement blood-clotting factor levels. Individuals with moderate to severe often remain at risk for spontaneous bleeding episodes despite prophylaxis and standard IV infusions.
The FDA based approval of fidanacogene elaparvovec-dzkt on results of the single-arm, phase 3 BENEGENE-2 study.
The study evaluated the safety and efficacy of the therapy for adult men with moderately severe to severe hemophilia B.
Annualized bleeding rate served as the study’s main objective.
Researcher enrolled 45 men (age range, 18 to 65 years) who completed a minimum 6 months of routine factor IX prophylaxis therapy during the lead-in study. The men received a single IV infusion of fidanacogene elaparvovec-dzkt dosed at 5 x 1011 vg/kg.
The study met its primary endpoint, showing noninferiority in terms of annualized bleeding rate after infusion compared with factor IX prophylaxis.
Researchers reported a mean annualized bleeding rate of 2.5 among men who received fidanacogene elaparvovec-dzkt in the evaluation period vs. a mean annualized bleeding rate of 4.5 during the lead-in pretreatment period.
Results showed a median zero (range, 0-19) annualized bleeding rate with fidanacogene elaparvovec-dzkt compared with a median rate of 1.3 (range, 0-53.9) with prophylaxis.
Fidanacogene elaparvovec-dzkt also appeared associated with a higher rate of bleed elimination than prophylaxis (60% vs. 29%).
The most common adverse event reported in trials of the therapy has been elevated transaminases. Twenty-six of 60 patients treated at the recommended dose had elevated transaminases, and 31 of 60 received corticosteroids. No serious adverse events and no deaths have been reported.
“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,” Adam Cuker, MD, MS, associate professor of medicine at Hospital of the University of Pennsylvania and director of the Penn Comprehensive and Hemophilia Thrombosis Program, said in the press release. “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.”
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