What is Elocta®

Elocta® is the first extended half-life recombinant factor VIII approved in the EU

Elocta® is indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). Elocta can be used for all age groups.1

Recommended dosing 

For long term prophylaxis, the recommended dose is 50 IU/kg every 3 to 5 days.1 The dose may be adjusted based on individual response in the range of 25-65 IU/kg.1 In some cases, especially in younger patients, shorter dosing intervals or higher doses may be necessary.1

  • Patients may be switched empirically with no need for extensive PK assessment2

 

Factor VIII activity graph

More time with factor VIII activity above baseline versus conventional factor VIII2

*Among patients (n=28) after a single dose (50 IU/kg) in the pivotal phase 3 trial. Presented as mean (±standard error). Baseline = pre-injection after a 96-hour washout. †Elocta® measured up to 120 hours, Advate measured up to 72 hours.

References

1. Elocta, Summary of Product Characteristics, 2017.

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

Extended half life

Longer time to 1 IU/dL and 3 IU/dL above baseline 

Elocta’s extended half-life provides patients with a longer time to 1 IU/dL (1%) and 3 IU/dL (3%) above baseline versus conventional factor VIII.

Advate vs Elocta - mean time to 1% above baseline. Advate™ 3.3 days - Elocta® 4.9 days
Advate vs Elocta - mean time to 3% above baseline. Advate™ 2.5 days - Elocta® 3.7 days

*Among patients (n=28) after a single dose (50 IU/kg) in the pivotal phase 3 trial. Presented as geometric mean (95% confidence intervals). Baseline = pre-injection after a 96-hour washout.

References

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

FC Fusion

Delaying degradation with Fc fusion

Elocta® is a recombinant fusion protein, composed of recombinant factor VIII covalently fused to the Fc domain of human immunoglobulin G1.1 The Fc portion of Elocta binds to neonatal Fc receptor (FcRn), which is part of a naturally occurring pathway that delays lysosomal degradation of Fc-containing proteins by cycling them back into circulation and thus extends their plasma half-life. 2,3

References

1. Dumont JA et al. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood 2012;119(13): 3024–30.
2. Roopenian DC, and Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 2007 Sep;7(9):715-25
3. Shapiro A. Development of long-acting recombinant FVIII and FIX Fc fusion proteins for the management of hemophiliaExpert Opin Biol Ther 2013;13(9):1287-97

Manufacturing process

Protecting the purity of Elocta®

Protection starts with the manufacturing of Elocta in world-class facilities and by incorporating state-of-the-art purification, filtration and viral inactivation steps.

Safety

Elocta® safety profile1,2

The adverse reaction frequencies in the table below were observed in a total of 233 patients with severe haemophilia A in phase 3 clinical studies and an extension study. The total number of exposure days was 34,746 with a median of 129 (range 1-326) exposure days per subject.

Frequencies have been evaluated according to the following convention: very common (≥1/10); common (≥1/100 to <1/10); uncommon (≥1/1,000 to <1/100); rare (≥1/10,000 to <1/1,000); very rare (<1/10,000), not known (cannot be estimated from the available data).

MedDRA System Organ Class

Adverse reactions

Frequency category

Blood and lymphatic system disorders

FVIII inhibition

Uncommon (PTPs)*

Nervous system disorders

Headache

Dizziness

Dysgeusia

Uncommon

Uncommon

Uncommon

Cardiac disorders

Bradycardia

Uncommon

Vascular disorders

Hypertension

Hot flush

Angiopathy

Uncommon

Uncommon

Uncommon

Respiratory, thoracic, and mediastinal disorders

Cough

Uncommon

Gastrointestinal disorders

Abdominal pain, lower

Uncommon

Skin and subcutaneous tissue disorders

Rash

Uncommon

Musculoskeletal and connective tissue disorders

Arthralgia

Myalgia

Back pain

Joint swelling

Uncommon

Uncommon

Uncommon

Uncommon

General disorders and administration site conditions

Malaise

Chest pain

Feeling cold

Feeling hot

Uncommon

Uncommon

Uncommon

Uncommon

Injury, poisoning, and procedural complications

Procedural hypotension

Uncommon

 

investigator term: vascular pain after injection of Elocta®
One adult subject had a positive anti-Factor VIII antibody test result coincident with a single measurement of a neutralising antibody titre of 0.73 Bethesda Units/mL at Week 14. The neutralising antibody was not confirmed upon repeat testing 18 days later and was negative at subsequent visits. There was an increase in clearance (CL) at Week 14 that resolved with continued rFVIIIFc treatment. 

For full information on safety and adverse events, please read the Elocta SmPC.

References

1. Elocta, Summary of Product Characteristics, 2017.
2. Mahlangu J et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014;123(3):317–25.