BeneFIX: For once-weekly prophylaxis (patients ≥16 years old) and on-demand use​​​​​​​2

On-Demand Bleed Control

PUPs

Indicated for bleed control in PUPs2

94.1% of responses were rated excellent or good in PUPs receiving an initial infusion of BeneFIX for on-demand treatment2

  • 2.9% of responses were rated moderate, 1% were rated as no response, 2% were not rated2,3.‡
  • 1505 infusions were given for on-demand treatment of 997 hemorrhages3
  • All bleeding episodes were controlled3

75% of bleeds were controlled after 1 infusion with BeneFIX3

  • 14.9% were controlled after 2 infusions
  • 5.4% were controlled after 3 infusions
  • 2.4% were controlled after 4 infusions
  • 2.2% were controlled after >4 infusions

In the same study,§ 84.3% of PUPs did not have spontaneous bleeding within 48 hours of their last infusion.2

   Study included 54 evaluable patients; median dose 62.7 IU/kg (range: 8.2-292 IU/kg).3

   Bleed control rating scale: Efficacy was rated by the caregiver or investigator using a 4-point scale, based on a comparison with previously used plasma-derived factor IX (pdFIX) products for similar bleeds or procedures: excellent (as much and as rapid an improvement as the best pdFIX-related responses), good (as much and as rapid an improvement as most pdFIX-related responses), moderate (not as good as most pdFIX-related responses), or no response (no improvement).3,4

   §Study included 32 evaluable patients; mean dose (± SD) 73.3 IU/kg (±33.1 IU/kg).2

Safety profile—supported by clinical trials​​​​​​​

2 out of 63 PUPs developed high-titer (>5 BU/dL) inhibitors in clinical trials.2,3

  • Both PUPs with high-titer inhibitors were withdrawn from the study2,3
  • The incidence of factor IX inhibitors is consistent with reports of hemophilia B patients treated with pdFIX or PTPs treated with recombinant factor IX3
  • 56 patients received a total of 5032 infusions (mean 89.86 ± 74.04) for a mean duration of exposure of 86.75 ± 71.73 days3

Adverse reactions (<5%) reported for PUPs2,||

Adverse reaction

Number of patients

Hives

3 (4.8%)

Dyspnea

2 (3.2%)

Injection site reaction

1 (1.6%)

Chills

1 (1.6%)

Rash

1 (1.6%)

    ||Adverse reactions were reported within 72 hours of an infusion of BeneFIX.2

Study description: A multicenter, international, open-label, single-cohort study consisting of a core phase and an extension phase. In the core phase, patients received recombinant factor IX (rFIX) for (1) on-demand treatment in response to any bleeding episode, (2) routine prophylaxis, or (3) surgical prophylaxis. Patients who completed the core phase were allowed to enter the extension phase to receive rFIX for up to 1 additional year, at the discretion of the investigator and after prior written authorization from the sponsor.2,3

PTPs

Indicated for bleed control in PTPs2

90.9% of responses were rated excellent or good in PTPs receiving an initial infusion of BeneFIX for on-demand treatment4

  • 7.1% were rated as moderate, 0.7% were rated as no response, 1.3% were not rated#
  • 2758 infusions were given for on-demand treatment of 1796 hemorrhages
  • All bleeding episodes were controlled

80.9% of bleeds were controlled after 1 infusion with BeneFIX4

  • 11.6% were controlled after 2 infusions
  • 3.6% were controlled after 3 infusions
  • 1.8% were controlled after 4 infusions
  • 2.1% were controlled after >4 infusions

In the same study,** 63% of PTPs did not have spontaneous bleeding within 48 hours of their last infusion.2

   Study included 55 evaluable patients; median dose 42.8 IU/kg (range: 6.5-224.6 IU/kg).4

   #Bleed control rating scale: Efficacy was rated by the patient or investigator using a 4-point scale, based on a comparison with previously used pdFIX products for similar bleeds or procedures: excellent (as much and as rapid an improvement as the best pdFIX-related responses), good (as much and as rapid an improvement as most pdFIX-related responses), moderate (not as good as most pdFIX-related responses), or no response (no improvement).4

​​​​ **Study included 19 evaluable patients; mean dose 40.3 IU/kg (range: 13-78 IU/kg).4

Safety in PTPs—low inhibitor formation

1 out of 65 patients developed a low-titer transient inhibitor in clinical trials including children and adults.2,†† 

Adverse reactions (>5%) reported for PTPs2,||:

Adverse reaction

Number of patients

Headache

7 (10.8%)

Dizziness

5 (7.7%)

Injection site reaction

5 (7.7%)

Nausea

4 (6.2%)

Injection site pain

4 (6.2%)

Rash

4 (6.2%)

 ††At study completion (approximately 15 months after inhibitor detection), the patient had normal factor IX recovery pharmacokinetics. Patient populations across these studies represented different hemophilia severities, prior rFIX replacement treatment histories, and ages.2,4

Study description: A 20-center international trial was conducted in PTPs with severe or moderate (<5 IU/dL factor IX activity) hemophilia B. Participants received rFIX for pharmacokinetic studies, treatment of or prophylaxis against hemorrhage, or surgical hemostasis, and were assessed at 3-month intervals for 2 years.4


References:
  1. Center for Biologics Evaluation and Research, US Food and Drug Administration, US Department of Health and Human Services. CBER Approval Letter, Coagulation Factor IX (Recombinant), Genetics Institute, Inc. February 11, 1997. Accessed May 12, 2021. wayback.archive-it.org/7993/20170723024338/https://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm057039.htm
  2. BeneFIX. Prescribing information. Pfizer Inc.; 2021.
  3. Shapiro AD, Di Paola J, Cohen A, et al; Recombinant Factor IX Study Group. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood. 2005;105(2):518-525. doi:10.1182/blood-2004-06-2283
  4. Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL; Recombinant Factor IX Study Group. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood. 2001;98(13):3600-3606. doi:10.1182/blood.v98.13.3600

Efficacy & Safety

  • Once-Weekly Prophylaxis
  • On-Demand Bleed Control
  • Surgical Efficacy
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  • BeneFIX is contraindicated in patients who have manifested life-threatening, immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including hamster protein (CHO).
  • Hypersensitivity reactions, including anaphylaxis, have been reported with BeneFIX. Closely monitor patients for signs and symptoms of acute anaphylaxis, particularly during the early phases of initial exposure to the product. Immediately discontinue the administration of the product and initiate appropriate treatment if symptoms occur.
  • Patients may develop hypersensitivity to hamster protein as BeneFIX contains trace amounts.
  • BeneFIX has been associated with the development of thromboembolic complications, including in patients receiving continuous infusion through a central venous catheter. The safety and efficacy of BeneFIX  administration by continuous infusion have not been established.
  • Neutralizing antibodies (inhibitors) have been reported following the administration of BeneFIX. If expected plasma factor IX activity levels are not attained, or if the patient presents with an allergic reaction, or if bleeding is not controlled following an expected dose of BeneFIX, perform an assay that measures factor IX inhibitor concentration.
  • The most common adverse reactions (>5%) from clinical trials were fever, cough, nausea, injection site reaction, injection site pain, headache, dizziness, and rash.

BeneFIX, Coagulation Factor IX (Recombinant), is a human blood coagulation factor indicated for the treatment of:

  • Adults and children with hemophilia B (congenital factor IX deficiency or Christmas disease) for:
    • On-demand treatment and control of bleeding episodes.
    • Perioperative management of bleeding.
  • Patients 16 years of age and older with hemophilia B (congenital factor IX deficiency or Christmas disease) for:
    • Routine prophylaxis to reduce the frequency of bleeding episodes.

Limitation of use:

BeneFIX is not indicated for induction of immune tolerance in patients with hemophilia B.

Please see full Prescribing Information for BeneFIX.

Indication

BeneFIX, Coagulation Factor IX (Recombinant), is a human blood coagulation factor indicated for the treatment of:

  • Adults and children with hemophilia B (congenital factor IX deficiency or Christmas disease) for:
    • On-demand treatment and control of bleeding episodes.
    • Perioperative management of bleeding.
  • Patients 16 years of age and older with hemophilia B (congenital factor IX deficiency or Christmas disease) for:
    • Routine prophylaxis to reduce the frequency of bleeding episodes.

Limitation of use:
BeneFIX is not indicated for induction of immune tolerance in patients with hemophilia B.

Please see full Prescribing Information for BeneFIX.