ELOCTA

Efmoroctocog alfa is a fully recombinant factor VIII-Fc fusion protein (rFVIIIFc) with an extended half-life compared with conventional factor VIII (FVIII) preparations, including recombinant FVIII (rFVIII) products such as Moroctocog alfa 1.

It is an antihemorrhagic agent used in replacement therapy for patients with haemophilia A (congenital factor VIII deficiency).

It is suitable for all age groups.

A is a rare bleeding disorder associated with a slow clotting process caused by the deficiency of factor VIII.

Patients with this disorder are more susceptible to recurrent bleeding episodes and excessive bleeding following minor traumatic injuries or surgical procedures 1.

Prophylactic treatment may dramatically improve the management of severe haemophilia A in the future by reducing joint bleeding and other hemorrhages that cause chronic pain and disability to patients 1, 2.

Prophylaxis has also shown to reduce the formation of neutralizing anti-FVIII antibodies, or inhibitors 2.

VIII is a blood coagulant factor involved in the intrinsic pathway to form fibrin, or a blood clot.

Efmoroctocog alfa is a first commercially available rFVIII-Fc fusion protein (rFVIIIFc) where the conjugated molecule of rFVIII to polyethylene glycol is covalently fused to the dimeric Fc domain of human immunoglobulin G1, a long-lived plasma protein Label.

The B domain of factor

VIII is deleted.

In animal models of haemophilia, efmoroctocog alfa demonstrated an approximately two-fold longer t½ than commercially available rFVIII products 1.

Other drug products with similar structure and function to Efmoroctocog alfa include Moroctocog alfa, which is produced by recombinant DNA technology and is identical in sequence to endogenously produced Factor VIII, but does not contain the B-domain, which has no known biological function, and Antihemophilic factor human, which is purified endogenous Factor VIII from human pooled blood and contains both A.

It is commonly marketed as Elocta or Eloctate for intravenous injection.

To date, no confirmed inhibitory autoantibodies were seen in previously treated patients included in clinical studies and treatment-emergent adverse events were generally consistent with those expected in the patient populations being studied 1.

The extended half-life of efmoroctocog alfa provides several clinical benefits for patients, including reduced frequency of injections required and improved adherence to prophylaxis 1.

Indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency) Label.

In two multinational, open-label, noncomparative phase III trials involving previously treated pediatric and adult patients with severe haemophilia A, the clinical efficacy and safety of efmoroctocog alfa have been studied.

The bleeding episodes were adequately controlled and bleeding rates were substantially reduced when efmoroctocog alfa has been used for individualized prophylaxis or treatment of bleeding Label.

In adult patients receiving a single preoperative dose to maintain haemostasis during surgical procedures, the total dose on the day of surgery needed to maintain haemostasis ranged from 50.8-126.6 IU/kg Label.

von Willebrand factor Binding.

Following a single intravenous dose of 50 IU/kg in previously-treated adult patients with severe haemophilia A, mean peak plasma concentrations (Cmax) ranged from 108-131 IU/dL.

Mean area under the

FVIII activity time curve (AUC/Dose) ranged from 47.5-51.2 IUxh/dL per IU/kg.

Following a single intravenous dose of 50 IU/kg in previously-treated adult patients with severe haemophilia A, mean volume of distribution at steady state (Vss) ranged from 49.1-52.6 mL/kg. Mean Vss in adolescent patients 12-18 years of age ranged from 57.6-59.4 mL/kg. Mean Vss in pediatric patients < 12 years of age ranged from 49.5-63.1 mL/kg Label.

There are no detectable metabolites for efmoroctocog alfa.

It is presumed to be metabolized via a same degradation pathway as endogenous factor VIII.

Following a single intravenous dose of 50 IU/kg in previously-treated adult patients with severe haemophilia A, mean half life (t1/2) ranged from 19-20.9 h.

Mean t1/2 in adolescent patients 12-18 years of age ranged from 16-17.5 h.

Mean t1/2 in pediatric patients < 12 years of age ranged from 12.3-15.9 h Label.

Following a single intravenous dose of 50 IU/kg in previously-treated adult patients with severe haemophilia A, mean clearance (CL) rate ranged from 1.95-2.11 mL/h/kg. Mean CL in adolescent patients 12-18 years of age ranged from 2.45-2.62 mL/h/kg. Mean t1/2 in pediatric patients < 12 years of age ranged from 2.61-3.86 mL/h/kg Label.

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Based on the findings from acute and repeated dose toxicity studies, efmoroctocog alfa displays no special hazard for humans.

Studies to assess the genotoxicity, carcinogenicity, toxicity to reproduction or embryo-foetal development of efmoroctocog alfa have not been conducted.

In a placental transfer study, efmoroctocog alfa has been shown to cross the placenta in small amounts in mice Label.