Efficacy and safety

Reliable efficacy when you and your patients need it most1–3

Prophylaxis

Prophylactic treatment in PTPs
of responses were rated as excellent or effective (n=47)1,*
Prophylactic treatment in PUPs
of responses were rated as excellent (n=42), indicating that spontaneous musculoskeletal bleeding was prevented2
Paediatric patients
of patients under 6 years old experienced no spontaneous bleeding episodes whilst on prophylaxis (n=22)5

*The response to secondary prophylaxis was assessed once every 3 months by the investigator using a 3-point scale: excellent (no spontaneous bleeding of any kind, no change in dosing regimen necessary); effective (no spontaneous musculoskeletal bleeding, no change in dosing regimen necessary); or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).1

Rated by the investigator at 3-month intervals on a 3-point scale: excellent (prophylaxis treatment completely prevented spontaneous musculoskeletal bleeding, no change in dosing regimen necessary); effective (adequately prevented spontaneous musculoskeletal bleeding episodes as demonstrated by a lower than expected incidence of spontaneous musculoskeletal bleeding episodes); or inadequate (inadequate prevention of bleeding requiring a change in dosing regimen).2

 

On-demand treatment

On-demand treatment in PTPs
of 1,796 bleeding episodes were resolved with only 1 infusion (n=55)1
On-demand treatment in PUPs
of 997 bleeding episodes were resolved with only 1 infusion (n=54)2
Surgery
of responses were rated excellent or good (n=28)3,§

Haemostasis was rated as excellent or good in 34 out of 35 of the operative procedures. Response ratings are defined as follows: excellent if the response was as satisfactory, with as much and as rapid an improvement as the best responses with other factor IX 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 factor IX products for similar bleeds or procedures; moderate if the response was less than satisfactory and not as good as most responses seen with other factor IX products when used for similar bleeding episodes or similar procedures; or no response if no improvement at all was observed.3

§ 29% (8/28) of patients (9 operative procedures) received postoperative BeneFIX® by continuous infusion.BeneFIX® is not licensed for use by continuous infusion – please refer to the BeneFIX® Summary of Product Characteristics for further information.4

 

Safety

A favourable safety profile

The most common adverse reactions reported in clinical trials of PTPs and identified in post-marketing use were4:

  • Headache, cough, pyrexia – very common (≥10%)
  • Hypersensitivity, dizziness, dysgeusia, phlebitis, flushing, vomiting, nausea, rash, urticaria, chest discomfort, infusion-site reaction, infusion-site pain – common (≥1% to <10%)

Low inhibitor formation

Data from more than 11,000 infusions of BeneFIX® demonstrate that inhibitor development and allergic reactions were low6,7:

  • Results of pooled safety data from 6 prospective studies showed that 1.2% of patients developed inhibitors on BeneFIX® (5/412)6
    • 4 of these patients also exhibited an allergic-type manifestation
  • Results from a retrospective study suggest that there is no difference in the frequency of allergic reactions or inhibitor development in patients receiving BeneFIX® versus those receiving plasma-derived factor IX concentrates7

 

References

1. Roth DA et al. 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.

2. Shapiro AD et al. 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.

3. Ragni MV et al. Use of recombinant factor IX in subjects with haemophilia B undergoing surgery. Haemophilia 2002;8(2):91–97.

4. Benefix® (nonacog alfa) Summary of Product Characteristics BF_16 : Accessed October 2020.

5. Monahan PE et al. Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 2010;16(3):460–468.

6. Rendo P et al. Nonacog alfa: an analysis of safety data from six prospective clinical studies in different patient populations with haemophilia B treated with different therapeutic modalities. Blood Coagul Fibrinolysis 2015;26(8):912–918.

7 Recht M et al. A retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia B. Haemophilia 2011;17(3):494–499.

PTP = previously treated patient; PUP = previously untreated patient.

PP-BEN-GBR-0418. October 2020