ELOCTATE

What it is

ELOCTATE is a long-acting, fully recombinant fusion protein comprised of recombinant B domain-deleted human factor VIII covalently linked to the Fc domain of human IgG1. Efmoroctocog alfa is produced by recombinant DNA technology in a human embryonic kidney cell line. ELOCTATE is formulated as a sterile, preservative-free, non-pyrogenic, lyophilised, white to off-white powder to cake, for intravenous administration in a single-use vial. Each single-use vial contains nominally 250, 500, 1000, 2000, or 3000 International Units of efmoroctocog alfa.

Approved indications

ELOCTATE is indicated in adults and children with haemophilia A (congenital factor VIII deficiency) for: — Control and prevention of bleeding episodes. — Routine prophylaxis to prevent or reduce the frequency of bleeding episodes. — Perioperative management (surgical prophylaxis).

Dosing overview

1 IU of ELOCTATE per kg body weight is expected to increase the circulating level of factor VIII by 2% [IU/dL]. For routine prophylaxis, the recommended regimen is 50 IU/kg every 3 to 5 days, with the dose adjustable based on patient response in the range of 25 to 65 IU/kg. For weekly prophylaxis, the recommended dose is 65 IU/kg. More frequent or higher doses up to 80 IU/kg may be required in children less than 12 years of age. For bleeding episodes and perioperative management, dosing varies by indication severity. For minor and moderate bleeds, the desired peak factor VIII level is 40 to 60 IU/dL, administered as 20 to 30 IU/kg and repeated every 24 to 48 hours until bleeding is resolved. For major bleeds, the desired peak factor VIII level is 80 to 100 IU/dL, administered as 40 to 50 IU/kg and repeated every 12 to 24 hours until bleeding is resolved. For minor surgery, the target factor VIII level is 50 to 80 IU/dL, with a single infusion of 25 to 40 IU/kg often sufficient and repeated every 24 hours as needed. For major surgery, the target factor VIII level is 80 to 120 IU/dL, with an initial preoperative dose of 40 to 60 IU/kg followed by 40 to 50 IU/kg after 8 to 24 hours and then every 24 hours to maintain activity within the target range.

Key safety warnings

Allergic type hypersensitivity reactions, including anaphylaxis, are possible with factor replacement therapies. Hypersensitivity reactions have been reported with ELOCTATE. Patients should discontinue use if hypersensitivity symptoms occur and seek immediate medical attention. Inhibitors have been reported with factor replacement therapy in the treatment of haemophilia A. Patients using ELOCTATE should be monitored for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests, including in previously untreated patients. In patients with existing cardiovascular risk factors, substitution therapy with factor VIII may increase the cardiovascular risk. If a central venous access device is required, risk of device-related complications including local infections, bacteraemia and catheter site thrombosis should be considered.

Contraindications

ELOCTATE is contraindicated in patients who have manifested severe hypersensitivity reactions, including anaphylaxis, to the product or its components.

Regulatory history

ELOCTATE efmoroctocog alfa was first listed on the ARTG on 27 June 2014 in five strengths: 250 IU, 500 IU, 1000 IU, 2000 IU, and 3000 IU, as powder for injection vials with diluent pre-filled syringes under licence category RE.