Why Elocta®

Elocta® is the only EHL FVIII with >3.5 years of real-world experience.*

*Claim based on the FDA approval date for rFVIIIFc (2014) and FVIII-rurioctacog alfa pegol (2016)

Those experiencing bleeds on prophylaxis could have fewer bleeds with Elocta® than with conventional factor VIII.1,2,3 This was a post-hoc analysis and not the original aim of the trial.
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Patients could resolve most bleeds with a single injection of Elocta®.1,3
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Patients on prophylaxis could have fewer injections with Elocta® than with conventional factor VIII.2,3
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References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.

2. Shapiro AD et al. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost. 2014;12:1788–800.

3. Young G et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe haemophilia A. J Thromb Haemost. 2015;13:967-77.

Fewer bleeds on prophylaxis

Patients on individualised prophylaxis with Elocta® experienced fewer bleeds than those on on-demand treatment with Elocta®1,2,3

Chart ABR by type
  • Median ABR (IQR) of 1.6 (0.0, 4.7) overall in adults and adolescents on individualised prophylaxis with Elocta®1
  • Median ABR of 0.0 in the last 3 months of the study (versus 6.0 with conventional factor VIII in the 12 months pre-study)*2
  • Median ABR (IQR) of 1.96 (0.00, 3.96) overall in children on prophylaxis with Elocta3 

*IQR data not available

References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.

2. Shapiro AD et al. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost. 2014;12:1788–800.

3. Young G et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015;13:967–77.

Reducing injections on prophylaxis

Almost all patients converting from conventional prophylaxis therapy reduced the number of injections on individualised prophylaxis with Elocta® in clinical trials1,2

Statistics Elocta vs conventional phylaxis medicin

99% of adults and adolescents needed fewer injections on individualised prophylaxis with Elocta compared with their pre-study regimen.1,2

References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.

2. Shapiro AD et al. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost. 2014;12:1788–800.

Resolving bleeds with one injection

Patients could resolve most bleeds with a single injection of Elocta®1,2

Chart: 87.3% of bleeds resolved with one injection in adults
  • Median dose/injection to treat a bleeding episode: 27.4 IU/kg (n=757)1
  • For bleeds that required more than one injection, median (IQR) interval between injections was 30.9 (24.5, 49.0) hours1
Chart: 81,7% of bleeds resolved with 1 injection in children
  • Median dose/injection to treat a bleeding episode: 49.7 IU/kg (n=86)2
  • Median total dose to treat a bleeding episode: 54.9 IU/kg (n=86)2

87.3% of bleeds in adolescents and adults and 81.4% in children were resolved with a single injection of Elocta1,2 

References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.

2. Young G et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015;13:967–77.

Efficacy in surgery

Haemostatic response in surgery 

Investigators rated haemostatic response as “Excellent” or “Good” in major surgical procedures in A-LONG.1,2

Efficacy in surgery
Efficacy in surgery

References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317-25.

2. Mahlangu J et al. Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) for perioperative haemostatic management in severe haemophilia A. Thromb Haemost. 2016 Jul 4;116(1):1-18.

Clinical trials summary

The Elocta clinical trial programme is one of the largest in haemophilia A.

  • A-LONG
  • Kids A-LONG
  • ASPIRE

A-LONG and data in adults

The A-LONG study demonstrated that Elocta was well tolerated and efficacious in the prevention and treatment of bleeding events in adolescents and adults with severe haemophilia A1

Chart showing information on A-long trail design
  • A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and pharmacokinetic properties of Elocta® in 165 previously treated males with severe haemophilia A1
  • The individualised prophylaxis regimen was adjusted to maintain trough level >1–3 IU/dL above baseline, as clinically indicated to prevent bleeding. Dose adjusted in a range of 25-65 IU/kg and interval of every 3-5 days1

References

1. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.

Kids A-LONG and data in children

The Kids A-LONG study demonstrated that twice-weekly injections with Elocta® was well tolerated and yielded low bleeding rates in children with severe haemophilia A.

A-long trail design for children

 

  • Kids A-LONG was an open-label, multinational, phase 3 trial evaluating the safety, efficacy and pharmacokinetic properties of Elocta in 71 previously treated children with severe haemophilia A1

References

1. Young G et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Haemost. 2015;13:967–77

ASPIRE

ASPIRE was a phase 3, open-label, multi-centre extension trial evaluating the long-term safety and efficacy of Elocta in subjects completing A-LONG and Kids A-LONG.

Aspire trial design for A-long and Kids A-long

Interim extension trial safety data is consistent with phase 3 trials.1 

References

1. Nolan et al. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016; 22:72-80.