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About BeneFix

What is BeneFix?

BeneFix is a recombinant clotting factor IX that temporarily
replaces the factor IX that is missing or at low levels in
people with hemophilia B

Designed with viral safety in mind

  • Not derived from human blood and contains no preservatives or added human or
    animal components
  • More than 150 quality control tests are done on each batch of BeneFix

BeneFix—the recombinant factor IX you've
turned to for more than 20 years*

Chinese hamster ovary (CHO) cells used to manufacture BeneFix are grown in media containing
no added human or animal components.

Bleed control and prevention

Demonstrated bleed control and prevention in previously
untreated patients (PUPs)

Indicated for bleed control and prevention§ in PUPs

94.1% of responses

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

  • 2.9% of responses were rated moderate, 1% of responses were rated no response,
    2% were not rated||
  • 1505 infusions were given for on-demand treatment of 997 hemorrhages
  • All bleeding episodes were controlled

75%of bleeds

75% of bleeds were controlled after 1 infusion with BeneFix

  • 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.

Study included 54 evaluable patients; median dose 62.7 IU/kg (8.2-292).
§Study included 32 evaluable patients; mean dose (±SD) 73.3 IU/kg (±33.1).
IIBleed 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).

STUDY DESCRIPTION FOR PREVIOUSLY UNTREATED PATIENTS

A multicenter, open-label, single-cohort study in which 54 PUPs were administered BeneFix (recombinant factor IX, rFIX) for (1) on-demand treatment in response to any bleeding episode (dose as deemed appropriate by the investigator), (2) routine prophylaxis for prevention of bleeding episodes, or (3) surgical prophylaxis. Patients who completed the core phase of the study 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 by the sponsor. This research was originally published in Blood.

Experience with previously treated patients (PTPs)

Indicated for bleed control and prevention# in PTPs

90.9%of responses

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

  • 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

80.9% of bleeds were controlled after 1 infusion with BeneFix

  • 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.

Study included 55 evaluable patients; median dose 42.8 IU/kg (range: 6.5-224.6 IU/kg).
#Study included 19 evaluable patients; mean dose 40.3 IU/kg (range: 13-78 IU/kg).
**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).

STUDY DESCRIPTION FOR PREVIOUSLY TREATED PATIENTS

A 20-center international trial was conducted in 56 previously treated patients 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 (for all interventions the regimen was prescribed by the investigator) for a mean duration of treatment of 23.4 ± 5.3 months and were assessed at 3-month intervals for 2 years. The research was originally published in Blood.

Safety

Low levels of inhibitors in BeneFix clinical trials

The body can make antibodies called “inhibitors.” These may stop BeneFix from working properly.
The higher the level of inhibitors, the more they may block effective treatment with BeneFix.

The strength of an inhibitor in a person’s blood is measured in Bethesda units (BU).

  • High titer (more than 5 BU)
  • Low titer (less than 5 BU)

In PUPs in clinical trials:

2

out
of

63

developed a high-titer
inhibitor.

Both patients were
removed from the study.

In PTPs in clinical trials including
children and adults:

1

out
of

65

developed a low-titer
inhibitor.††

††This patient completed the study and had
normal factor IX recovery (about 15 months
after the inhibitor was detected).

Dosing and R2 kit

Live actively? Talk to your doctor about a BeneFix dose
that’s right for you

BeneFix offers dosing flexibility with 5 vial sizes

  • 250 IU
  • 500 IU
  • 1000 IU
  • 2000 IU
  • 3000 IU

Flexibility and convenience:

BeneFix Rapid Reconstitution (R2) Kit

  • Range of vial sizes
  • Travel-ready pack
  • Single-use vial
  • Sterile infusion set
  • Environmentally friendly
    packaging means less waste

BeneFix can be stored at room temperature or under refrigeration
(2°C to 30°C/36°F to 86°F) for up to 2 years, until expiration.