Efficacy and Safety Data

Indicated for bleed control^† and prevention^‡ in PUPs²

^†Study included 54 evaluable patients; median dose 62.7 IU/kg (8.2-292).^3
‡Study included 32 evaluable patients; mean dose (± SD) 73.3 IU/kg (±33.1).²

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, 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 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

Indicated for bleed control^|| and prevention^¶ in PTPs²

^||Study included 55 evaluable patients; median dose 42.8 IU/kg (range: 6.5-224.6 IU/kg).^4
¶Study included 19 evaluable patients; mean dose 40.3 IU/kg (range: 13-78 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).^3,4

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,#

Most common adverse reactions (>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 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 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

Management of hemostasis in surgical procedures

28 PTPs underwent 36 surgeries^2,‡‡

23 PUPs underwent 30 surgeries^2,‡‡

Dental and minor procedures²

Surgical management 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.^3,4

^**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

^††In the PTP surgery subjects, the median increase in circulating factor IX activity was 0.7 IU/dL per IU/kg infused (range 0.3-1.2 IU/dL; mean 0.8 ± 0.2 IU/dL per IU/kg).^2
‡‡In the PUP surgery subjects, the mean dose per infusion was 89.87 ± 51.95 IU/kg, and mean treatment duration of treatment was 5.09 ± 3.96 days.³

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