Efficacy and Safety Data

BeneFix — a recombinant factor IX with data to support bleed prevention in previously untreated patients (PUPs)

Bleed control in PUPs

Safety profile—supported by clinical trials

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

Adverse reactions (<5%) reported for PUPs^2§

^§Adverse reactions were reported within 72 hours of an infusion of BeneFix.²

Study description: A multicenter, 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 for prevention of bleeding episodes, 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

Bleed prevention in previously treated patients (PTPs)

Bleed control in PTPs

Safety in PTPs—low inhibitor formation

1 out of 65 patients developed a low-titer transient inhibitor.^2#

Most common adverse reactions (frequency >5%) were²:

^#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 factor IX 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 recombinant factor IX for pharmacokinetic studies, treatment of or prophylaxis against hemorrhage, or surgical hemostasis, and were assessed at 3-month intervals for 2 years.⁴

Effective hemostasis in surgical procedures

28 PTPs underwent 36 surgeries²

23 PUPs underwent 30 surgeries²

Dental and minor procedures²

Rating scale: Efficacy was graded by subjective assessment of response to surgery by the subject and by the physician, in consultation with the surgeon. The response was rated excellent if the response was as satisfactory, with as much and as rapid an improvement as the best responses with other FIX products for similar bleeds or procedures; good if the response was as satisfactory, with as much and as rapid an improvement as most responses with other FIX products for similar bleeds or procedures; moderate if the response was less than satisfactory and not as good as most responses seen with other FIX products when used for similar bleeding episodes or similar procedures; or no response if no improvement at all was observed.⁵

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

PUPs: A multicenter, open-label, single-cohort study consisting of a core phase and an extension phase. In the core phase, patients received rFIX for: (1) on-demand treatment in response to any bleeding episode, (2) routine prophylaxis for prevention of bleeding episodes, 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

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