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About BeneFix

What is BeneFix (coagulation factor IX [recombinant])?

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.

Proven bleed protection with once-weekly dosing

Demonstrated low median annualized bleeding rate (ABR)

In a clinical study, patients on BeneFix once-weekly prophylaxis (100 IU/kg) had a lower median total ABR
than patients treated on demand.


Protection in patients using BeneFix once weekly (median study results):

2

Total ABR with BeneFix

(vs 34 with
on-demand treatment)

1

Annualized
spontaneous bleeding rate§

(vs 22 with
on-demand treatment)

0

ABR in joints

(vs 29 with
on-demand treatment)



STUDY DESCRIPTION

In a multicenter open-label clinical trial, 25 previously treated patients (PTPs) were treated with BeneFix administered at 100 IU/kg once weekly and evaluated for approximately 52 weeks. Common (≥5%) adverse reactions were headache (36%), fever (20%), and cough (8.0%). In the study, No patients were withdrawn from the study due to an adverse reaction, no inhibitors were detected, and no thrombotic (blood clotting) events or anaphylactic events were reported.


Proven bleed control

Demonstrated bleed control in previously untreated patients (PUPs)

Indicated for bleed control|| 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

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

STUDY DESCRIPTION FOR PUPs

A multicenter, international, 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, (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.

Experience with previously treated patients (PTPs)

Indicated for bleed control# 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).
**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 PTPs

A 20-center international trial was conducted in 56 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 (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)

For once-weekly prophylaxis

In PTPs in a clinical trial:

Zero patients

developed an inhibitor with BeneFix once weekly.

Patients included in the study had 100 exposure days to factor IX products and no current or past history of factor IX inhibitors prior to enrollment (N=25).

For on-demand treatment

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

BeneFix offers dosing options to meet your needs

Once-weekly prophylaxis or on-demand use

  • The recommended regimen for once-weekly prophylaxis is 100 IU/kg
  • Talk to your doctor about a BeneFix dose that’s right for you
  • Your doctor may adjust the amount or frequency of your dose based on your individual response

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

BeneFix can be stored at room temperature or under refrigeration
(2°C-30°C/36°F-86°F) for up to 2 years, until expiration. Do not freeze the BeneFix Kit.