Beqvez^™ (fidanacogene elaparvovec-dzkt – Pfizer) received approval from the U.S. Food and Drug Administration on April 25, 2024. It is a gene therapy to treat moderate to severe hemophilia B for patients who are 18 years old and older. Candidates for its use may need blood clotting factor IX (FIX) prophylaxis therapy, have current or historical life-threatening hemorrhaging, or have repeated, serious spontaneous bleeding episodes. They also must test negative for neutralizing antibodies to the adeno-associated viral vector used in Beqvez. It is administered as one intravenous (IV) infusion containing 5×10¹¹ vector genomes(vg)/kg. The launch will be during the second quarter of 2024 at a wholesale acquisition cost (WAC) of $3.5 million. Complete prescribing information is here.

At a Glance

• Brand (Generic) Name: Beqvez (fidanacogene elaparvovec-dzkt)
• Manufacturer: Pfizer
• Date Approved: April 25, 2024
• Indication: to treat adults who have moderate to severe hemophilia B, who currently use FIX prophylaxis therapy, who have current or historical life-threatening hemorrhage, or who have repeated, serious spontaneous bleeding episodes. They cannot have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
• Dosage Forms Available: kits of between four and seven vials with each containing at least 1mL of suspension at 1×10¹³ genome copies — for a weight-based dose of 5×10¹¹ vector genomes(vg)/kg for each patient.
• Launch Date: Q2:2024
• Estimated Cost: $3.5 million
• An inherited bleeding disorder, hemophilia B is caused by mutations in the F9 genes on X chromosomes. Patients who have it produce either insufficient or abnormal FIX, which leads to bleeding episodes that cause intense pain, permanent joint damage, and potentially life-threatening hemorrhages.
• In the United States, hemophilia B affects approximately 4,000 people, including some females. About one-third of patients have no family history of the condition.
• Beqvez is manufactured in a laboratory. After filtration, purification, and sterilization, the genetic material of bioengineered non-infective viral cells is replaced with functional FIX genes. The resulting combination is mixed with a promoter agent that targets human liver cells and then infused into eligible patients to deliver working copies of the enhanced FIX gene.
• In the phase III BENEGENE-2 clinical trial, the 45 patients, who all had FIX levels of 2% or less, had used their standard treatments for six months or longer before being given an infusion of Beqvez. In addition to reducing the annualized bleeding rate (ABR), another study goal was a final FIX level of 5% or more. Compared to an ABR average of 4.4 bleeds previous to the Beqvez infusion, the average ABR during the 12 months after the infusion was 1.3 and the need to treat spontaneous bleeds dropped by 78%. Average FIX levels were approximately 25% by 24 months resulting in a 92% decrease in the amount of FIX infusions needed by the patients.
• Although seven patients in the BENEGENE-2 trial experienced side effects that were considered to be serious, only two, a hemorrhaging ulcer and anemia, were determined to be associated with the therapy.
• None of the most frequently reported side effects were attributed to treatment with Beqvez.
• Including upper respiratory infections, allergic rhinitis, arthritis, and gastroesophageal reflux, all were mild and temporary.
• CSL Behring’s Hemgenix^® (etranacogene dezaparvovec-drlb) suspension is another gene therapy that was approved for hemophilia B by the FDA in late 2022. It also replaces mutated FIX genes to treat adults who use FIX prophylaxis therapy, who have had severe hemorrhages, or who have recurring, serious bleeds due to hemophilia B. It is administered as a single IV infusion. Its cost also is $3.5 million.