FAQ

Healthcare professionals frequently asked questions

1. For which patients can I use Elocta®?

Elocta® is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A and can be used for all age groups. 

The efficacy and safety of Elocta have been established in two phase 3, controlled trials: A-LONG and Kids A-LONG. 

A-LONG evaluated the safety, efficacy, and pharmacokinetics of Elocta use in 165 males who were at least 12 years of age and who had previously been treated with FVIII product.1 Patients with severe haemophilia A (defined as <1 IU/dL [<1%] endogenous FVIII activity) were treated with Elocta on-demand (n=23) or prophylactically (weekly [n=24] or individualised [n=118] prophylaxis regimen). 

Kids A-LONG evaluated the safety, efficacy and pharmacokinetics of Elocta, in previously treated children younger than 12 years old with severe haemophilia A.2 The study enrolled 71 subjects. The starting Elocta regimen was twice-weekly prophylaxis which was adjusted as needed.

The safety and efficacy of Elocta in previously untreated patients have not yet been established. Studies are ongoing.

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(6):967–977.

2. How do I convert a patient from conventional therapy to Elocta®?

How and when to switch patients are up to the discretion of the treating physician and the patient. According to the SmPC, treatment with Elocta® depends on the severity of the Factor VIII deficiency, the location and extent of bleeding, and the patient’s pharmacokinetic profile (e.g., half-life, in vivo recovery), and individual clinical response. 

  • Information about Dosing
  • For more detailed information about dosing and frequency of administration, see SmPC sections 4.2 and 5.2 respectively.

3. What about Elocta® and patient safety?

Elocta® is manufactured in world-class facilities incorporating state-of-the-art purification, filtration and viral inactivation steps. Elocta is produced by recombinant DNA technology in a human embryonic kidney (HEK) cell line without the addition of any exogenous human- or animal-derived protein in the cell culture process, purification or final formulation.

The safety, efficacy, and pharmacokinetics of Elocta were evaluated in two multinational, open-label, pivotal studies; a phase 3 study (A-LONG) in adolescents and adults and a phase 3 paediatric study (Kids-A-LONG) in children less than 12 years of age.1,2 No age-specific differences in adverse reactions were observed between paediatric and adults subjects. No patients developed inhibitors in the A-LONG and Kids A-LONG studies.* The reported adverse events (AEs) experienced in the studies were found to be consistent with those in the general haemophilia A population. No serious adverse events (SAEs) experienced were assessed as being related to Elocta use by the Investigators.

*Patients in these studies had previously been treated with conventional factor VIII.  As with other factor VIII products, inhibitor development has been observed in clinical practice.

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(6):967–977.

4. How can I be sure that Sobi is able to maintain an adequate supply of Elocta®?

Elocta® is currently manufactured by Sobi's collaboration partner Biogen at Research Triangle Park (North Carolina) and Cambridge (Massachusetts). Sobi understands how important it is for patients to have uninterrupted supply and is committed to providing sufficient supply of rFVIIIFc for both the participants enrolled in the haemophilia clinical programs and the market. Sobi believes that the current manufacturing capacity will provide ample supply for clinical programs, commercial demand and the donation programme.

5. How do I administer and reconstitute Elocta®

You will find instructions on how to administer and reconstitute Elocta® here

6. Any other questions?

Please contact Medical information for any other questions