|
| Primary information |
|---|
| ID | 10866 |
| Therapeutic ID | Th1227 |
| Protein Name | Efmoroctocog alfa |
| Sequence | NA
|
| Molecular Weight | 220000 |
| Chemical Formula | C9736H14863N2591O2855S78 |
| Isoelectric Point | NA |
| Hydrophobicity | NA |
| Melting point | NA |
| Half-life | 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 to 20.9 h. Mean t1/2 in adolescent patients 12 to 18 years of age ranged from 16 to 17.5 h. Mean t1/2 in pediatric patients < 12 years of age ranged from 12.3 to 15.9 h |
| Description | Efmoroctocog alfa is a long-acting, fully-recombinant factor VIII Fc fusion protein. |
| Indication/Disease | For the treatment of Haemophilia A. |
| Pharmacodynamics | NA |
| Mechanism of Action | Patients with haemophilia A lack factor VIII, a protein needed for normal clotting of the blood, and as a result, they bleed readily. The active substance in Elocta, efmoroctocog alfa, works in the body in the same way as human factor VIII. It replaces the missing factor VIII, thereby helping the blood to clot and giving temporary control of bleeding. |
| Toxicity | 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 [FDA Label]. |
| Metabolism | There are no detectable metabolites for efmoroctocog alfa. It is presumed to be metabolized via a same degradation pathway as endogenous factor VIII. |
| Absorption | 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 to 131 IU/dL. Mean area under the FVIII activity time curve (AUC/Dose) ranged from 47.5 to 51.2 IUxh/dL per IU/kg. Mean AUC/Dose in adolescent patients 12 to 18 years of age ranged from 38.2 to 40.8 IUxh/dL per IU/kg. Mean AUC/Dose in pediatric patients < 12 years of age ranged from 25.9 to 38.4 IUxh/dL per IU/kg [FDA Label]. |
| 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 to 52.6 mL/kg. Mean Vss in adolescent patients 12 to 18 years of age ranged from 57.6 to 59.4mL/kg. Mean Vss in pediatric patients < 12 years of age ranged from 49.5 to 63.1 mL/kg [FDA Label]. |
| Clearance | 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 to 2.11 mL/h/kg. Mean CL in adolescent patients 12 to 18 years of age ranged from 2.45 to 2.62 mL/h/kg. Mean t1/2 in pediatric patients < 12 years of age ranged from 2.61 to 3.86 mL/h/kg [FDA Label]. |
| Categories | Hemostatics |
| Patents Number | NA |
| Date of Issue | NA |
| Date of Expiry | NA |
| Drug Interaction | No interactions of human coagulation factor VIII (rDNA) with other medicinal products have been reported. |
| Target | von Willebrand factor |
| Brand Name | ELOCTA |
| Company | NA |
| Brand Description | NA |
| Prescribed For | Treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency). |
| Chemical Name | NA |
| Formulation | 0.6 mmol (or 14 mg) sodium per vial |
| Physical Appearance | Powder and solvent for solution for injection |
| Route of Administration | Intravenous |
| Recommended Dosage | The calculation of the required dose of recombinant factor VIII Fc is based on the empirical finding that 1 International Unit (IU) factor VIII per kg body weight raises the plasma factor VIII activity by 2 IU/dL. The required dose is determined using the following formula: Required units = body weight (kg) x desired factor VIII rise (%) (IU/dL) x 0.5 (IU/kg per IU/dL) |
| Contraindication | Hypersensitivity to the active substance (recombinant human coagulation factor VIII, and/or Fc domain) |
| Side Effects | Hypersensitivity or allergic reactions (which may include swelling of the face, rash, hives, tightness of the chest and difficulty breathing, burning and stinging at the infusion site, chills, flushing, generalised urticaria, headache, hypotension, lethargy, nausea, restlessness, tachycardia) have been observed rarely and may in some cases progress to severe anaphylaxis. |
| Useful Link 1 | Link |
| Useful Link 2 | NA |
| Remarks | NA |