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Unmet NeedsEfficacyEfficacyStudy DesignsResultsSafety ProfileClinical ScienceClinical ScienceHow BEQVEZ WorksFactor Levels Over TimeVector DNA ClearanceTreating with BEQVEZTreating with BEQVEZTreatment ConsiderationsPatient ProfilesPatient SelectionScreening for Neutralizing AntibodiesDosing ConsiderationsMonitoringResourcesResourcesSupport Services and AccessGetting Ready for BEQVEZDownloads & VideosFAQs
Full Prescribing InformationPatient InformationIndicationPatient Site

BEQVEZ is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and, do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

 Discover BEQVEZDiscover BEQVEZA one-time infusion designed
to evolve the treatment of
hemophilia B1A one-time infusion designed to evolve the treatment of hemophilia B1

A one-time infusion designed to help eligible patients make their own factor IX (FIX).* Duration of response is being evaluated in ongoing studies.1

A one-time infusion designed to help eligible patients make their own factor IX (FIX).* Duration of response is being evaluated in ongoing studies.1 

Safety studied since 2017—the longest clinical development program in any approved hemophilia B gene therapy.2-5†

BEQVEZ is a gene therapy designed to introduce in the transduced cells a functional copy of the FIX gene encoding a high-activity FIX variant. The AAVRh74var capsid is able to transduce hepatocytes, the natural site of FIX synthesis.1BEQVEZ was approved based on the pivotal Phase 3 study which was designed to assess the efficacy and safety of BEQVEZ. In a Phase 1/2 study and subsequent Phase 2a long-term follow-up (LTFU) study, participants received an infusion of BEQVEZ with the same vector construct, transgene, and dose of that which was used in the pivotal Phase 3 study. The Phase 1/2 study and subsequent Phase 2a LTFU study were designed to assess the safety of BEQVEZ—5 patients were studied up to Year 6.1 Discover what BEQVEZ may do for your patients Loading Discover what BEQVEZ may do for your patients Loading Treatment needs in hemophilia BPatients may still experience challenges with the current standard of care in hemophilia B6Understand patients' unmet needsLoadingHow BEQVEZ WorksBEQVEZ mechanism of actionLearn about the scienceLoading Discover what BEQVEZ may do for your patients Loading Discover what BEQVEZ may do for your patients Loading Treatment needs in hemophilia BPatients may still experience challenges with the current standard of care in hemophilia B6
Understand patients' unmet needs
LoadingHow BEQVEZ WorksBEQVEZ mechanism of actionLearn about the scienceLoadingSafetySafety studied since 2017—the
longest clinical development
program in any approved
hemophilia B gene therapy2-5†Explore safety profileLoadingPrepare to TreatSupport is here for your patients
and treatment center every step of
the waySee testing requirementsLoadingStay InformedAt Pfizer, we are committed to ensuring you have
the latest information about BEQVEZAt Pfizer, we are committed to ensuring you have 
the latest information about BEQVEZSign up to stay informedLoadingReferences:BEQVEZ. Prescribing Information. Pfizer Inc.; 2024.Clinicaltrials.gov. Accessed April 10, 2024. https://clinicaltrials.gov/study/NCT03307980Clinicaltrials.gov. Accessed April 10, 2024. https://clinicaltrials.gov/study/NCT03861273Clinicaltrials.gov. Accessed April 10, 2024. https://clinicaltrials.gov/study/NCT03489291Clinicaltrials.gov. Accessed April 10, 2024. https://clinicaltrials.gov/study/NCT03569891Srivastava A, Santagostino E, Dougall A, et al. Haemophilia. 2020;26(suppl 6):1-158.

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INDICATION
BEQVEZ is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:
  • Currently use factor IX prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleeding episodes, and,
  • Do not have neutralizing antibodies to adeno-associated virus serotype 
 Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
Select patients for therapy based on an FDA-approved companion diagnostic for BEQVEZ.
Important Safety InformationIntravenous administration of a liver-directed AAV vector could potentially lead to liver transaminase elevations. Monitor ALT, AST, and factor IX activity levels once or twice weekly for at least 4 months and institute corticosteroid treatment in response to transaminase elevation and/or decrease in factor IX activity, as required. Perform regular liver ultrasounds (i.e., annually) and alpha-fetoprotein (AFP) testing in patients with risk factors of hepatocellular carcinoma. Monitor for and manage adverse reactions secondary to corticosteroid therapy.

For the first year after BEQVEZ infusion, advise patients to limit alcohol consumption, which may impact liver enzyme elevation and reduce factor IX activity.

Infusion reactions, including hypersensitivity reactions and anaphylaxis, may occur. Closely monitor patients for clinical signs and symptoms of infusion reactions throughout the infusion period and for at least 3 hours after end of infusion. In the event of an infusion reaction, the infusion may be slowed or stopped. If the infusion is stopped, restart at a slower rate when the infusion reaction has resolved.

The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. Integration of AAV vector DNA into the host cell DNA in other tissues may also occur.

Monitor patients for factor IX activity over time, particularly during the timeframe for corticosteroid treatment decision making, and for inhibitors to factor IX if bleeding is not controlled or plasma factor IX activity levels decrease.

The most common adverse reaction (incidence ≥5%) reported in clinical studies was an increase in transaminases. 62% of patients in the phase 3 study received corticosteroids for transaminase elevation and/or decline in factor IX activity.

Use of hepatotoxic medicinal substances may reduce the efficacy of BEQVEZ, and the risk of serious hepatic reactions may increase following administration.

Prior to BEQVEZ administration, the patient’s existing medications should be reviewed to determine if they should be modified to prevent anticipated interactions. Monitor concomitant medications after BEQVEZ administration and evaluate the need to change concomitant medication based on patient’s hepatic status and risk.

Prior to BEQVEZ infusion, ensure patients are up to date on their vaccinations. If concomitant corticosteroid administration is needed following BEQVEZ infusion, delay administration of live vaccines until the patient has been weaned off corticosteroids.

BEQVEZ is not intended for administration in women.

No studies in animals or clinical studies have been performed to evaluate the potential effects of BEQVEZ on fertility in humans. Vector DNA was shed in semen but declined to undetectable levels in semen within a mean of 1 to 4 months after infusion. Male patients should refrain from donating sperm, be abstinent or use a male condom for up to 6 months after receiving BEQVEZ.

Thoroughly assess liver health and test for pre-existing neutralizing antibodies prior to administering BEQVEZ. Do not administer BEQVEZ to patients with a positive test or a prior history for factor IX inhibitors.

Patients who receive BEQVEZ should be enrolled in a 15-year registry to evaluate the long-term efficacy and safety of hemophilia treatments.

Please see full Prescribing Information for BEQVEZ.Indication

BEQVEZ is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:

  • Currently use factor IX prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleeding episodes, and,
  • Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

Select patients for therapy based on an FDA-approved companion diagnostic for BEQVEZ.