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Th1227 details
Primary information
ID10866
Therapeutic IDTh1227
Protein NameEfmoroctocog alfa
SequenceNA
Molecular Weight220000
Chemical FormulaC9736H14863N2591O2855S78
Isoelectric PointNA
HydrophobicityNA
Melting pointNA
Half-lifeFollowing 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
DescriptionEfmoroctocog alfa is a long-acting, fully-recombinant factor VIII Fc fusion protein.
Indication/DiseaseFor the treatment of Haemophilia A.
PharmacodynamicsNA
Mechanism of ActionPatients 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.
ToxicityBased 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].
MetabolismThere are no detectable metabolites for efmoroctocog alfa. It is presumed to be metabolized via a same degradation pathway as endogenous factor VIII.
AbsorptionFollowing 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].
ClearanceFollowing 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].
CategoriesHemostatics
Patents NumberNA
Date of IssueNA
Date of ExpiryNA
Drug InteractionNo interactions of human coagulation factor VIII (rDNA) with other medicinal products have been reported.
Targetvon Willebrand factor
Brand NameELOCTA
CompanyNA
Brand DescriptionNA
Prescribed ForTreatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency).
Chemical NameNA
Formulation0.6 mmol (or 14 mg) sodium per vial
Physical Appearance Powder and solvent for solution for injection
Route of AdministrationIntravenous
Recommended DosageThe 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)
ContraindicationHypersensitivity to the active substance (recombinant human coagulation factor VIII, and/or Fc domain)
Side EffectsHypersensitivity 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 1Link
Useful Link 2NA
RemarksNA