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Identification
- Summary
Efmoroctocog alfa is a recombinant Factor VIII used to treat and prevent bleeding in hemophilia A.
- Brand Names
- Eloctate
- Generic Name
- Efmoroctocog alfa
- DrugBank Accession Number
- DB11607
- Background
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 alfa1. 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. Haemophilia 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.
Factor 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- and B-subunits.
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.
- Type
- Biotech
- Groups
- Approved, Investigational
- Biologic Classification
- Protein Based Therapies
Blood factors / Fusion proteins / Peptides - Protein Chemical Formula
- C9736H14863N2591O2855S78
- Protein Average Weight
- 220000.0 Da (Apparent, B-domain deleted)
- Sequences
- Not Available
- Synonyms
- Antihemophilic Factor (Recombinant BDD), FC Fusion Protein
- Antihemophilic factor (recombinant, FC fusion protein)
- Coagulation factor VIII recombinant immunoglubulin g1 fusion protein
- Efmoroctocog alfa
Pharmacology
- Indication
Indicated for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency) Label.
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Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Management of Bleeding •••••••••••• Prophylaxis of Bleeding •••••••••••• Management of Perioperative bleeding •••••••••••• - Contraindications & Blackbox Warnings
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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 to 126.6 IU/kg Label.
- Mechanism of action
Factor VIII exists in a circulating protein complex consisting of two molecules via a non-covalent binding interaction; Factor VIII and von Willebrand factor. This complex remains inactive until the coagulation cascade is initiated, which activated factor VIII. Factor VIII is released from the protein complex upon activation and acts as a cofactor for factor IX-mediated conversion of factor X to activated factor X on phospholipid surfaces. Activated factor X is critical in converting prothrombin into thrombin and sequentially, thrombin converts fibrinogen to fibrin for the formation of a blood clot Label.
Haemophilia A is a X-linked hereditary disorder of blood coagulation due to decreased levels of functional factor. The disorder can lead to various disabling complications including bleeding into joints, muscles or internal organs, either spontaneously or as a result of accidental or surgical trauma Label. Efmoroctocog alfa is a recombinant fusion protein comprised of a single molecule of B-domain deleted human coagulation factor VIII covalently linked to the Fc domain of human immunoglobulin G1. It acts as a replacement therapy to increase the plasma levels of factor VIII, thereby enabling a temporary correction of the factor deficiency and correction of the bleeding tendencies Label.
Extended half-life of efmoroctocog alfa relative to endogenous factor VIII is explained by the Fc region binding to the neonatal Fc receptor expressed throughout life; the receptor is part of a naturally occurring pathway that protects immunoglobulins (and Fc fusion proteins) from lysosomal degradation by cycling them back into the circulation Label,1.
Target Actions Organism Avon Willebrand factor bindingHumans - 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 Label.
- Volume of distribution
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 Label.
- Protein binding
Like endogenous factor VIII, efmoroctocog alfa binds to von Willebrand factor in the circulation.
- Metabolism
There are no detectable metabolites for efmoroctocog alfa. It is presumed to be metabolized via a same degradation pathway as endogenous factor VIII.
- Route of elimination
Not Available
- 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 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 Label.
- Adverse Effects
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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.
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- No interactions found.
Products
Drug product information from 10+ global regionsOur datasets provide approved product information including:dosage, form, labeller, route of administration, and marketing period.Access drug product information from over 10 global regions.- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Elocta 750 IU Intravenous SWEDISH ORPHAN BIOVITRUM AB (PUBL) 2021-01-12 Not applicable EU 
Elocta 1500 IU Intravenous SWEDISH ORPHAN BIOVITRUM AB (PUBL) 2021-01-12 Not applicable EU Elocta 500 IU Intravenous SWEDISH ORPHAN BIOVITRUM AB (PUBL) 2021-01-12 Not applicable EU Elocta 4000 IU Intravenous SWEDISH ORPHAN BIOVITRUM AB (PUBL) 2021-01-12 Not applicable EU Elocta 1000 IU Intravenous SWEDISH ORPHAN BIOVITRUM AB (PUBL) 2021-01-12 Not applicable EU
Categories
- Drug Categories
- Chemical TaxonomyProvided by Classyfire
- Description
- Not Available
- Kingdom
- Organic Compounds
- Super Class
- Organic Acids
- Class
- Carboxylic Acids and Derivatives
- Sub Class
- Amino Acids, Peptides, and Analogues
- Direct Parent
- Peptides
- Alternative Parents
- Not Available
- Substituents
- Not Available
- Molecular Framework
- Not Available
- External Descriptors
- Not Available
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- 7PCM518YLR
- CAS number
- 1270012-79-7
References
- General References
- Frampton JE: Efmoroctocog Alfa: A Review in Haemophilia A. Drugs. 2016 Sep;76(13):1281-1291. doi: 10.1007/s40265-016-0622-z. [Article]
- Tiede A: Half-life extended factor VIII for the treatment of hemophilia A. J Thromb Haemost. 2015 Jun;13 Suppl 1:S176-9. doi: 10.1111/jth.12929. [Article]
- FDA Approved Drug Products: Eloctate (Antihemophilic Factor (Recombinant), Fc Fusion Protein) powder for intravenous injection [Link]
- External Links
- PubChem Substance
- 347911217
- 1543095
- Wikipedia
- Efmoroctocog_alfa
- FDA label
- Download (557 KB)
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample data4 Completed Prevention Hemophilia A 1 somestatus stop reason just information to hide 4 Completed Treatment Hemophilia A 1 somestatus stop reason just information to hide 4 Completed Treatment Hemophilia A With Inhibitors 1 somestatus stop reason just information to hide 4 Recruiting Treatment Factor VIII (FVIII) / Hemophilia A 1 somestatus stop reason just information to hide 3 Completed Other Severe Hemophilia A 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Injection, powder, for solution Intravenous; Parenteral 1000 IU Injection, powder, for solution Intravenous; Parenteral 1500 IU Injection, powder, for solution Intravenous; Parenteral 2000 IU Injection, powder, for solution Intravenous; Parenteral 250 IU Injection, powder, for solution Intravenous; Parenteral 3000 IU Injection, powder, for solution Intravenous; Parenteral 500 IU Injection, solution 1000 i.u. Injection, solution 1500 i.u. Injection, solution 2000 i.u. Injection, solution 250 i.u. Injection, solution 3000 i.u. Injection, solution 500 i.u. Kit Intravenous 1000 [iU]/3mL Kit Intravenous 1500 [iU]/3mL Kit Intravenous 2000 [iU]/3mL Kit Intravenous 250 [iU]/3mL Kit Intravenous 3000 [iU]/3mL Kit Intravenous 4000 [iU]/3mL Kit Intravenous 500 [iU]/3mL Kit Intravenous 5000 [iU]/3mL Kit Intravenous 6000 [iU]/3mL Kit Intravenous 750 [iU]/3mL Kit; powder, for solution Intravenous 1000 unit / vial Kit; powder, for solution Intravenous 1500 unit / vial Kit; powder, for solution Intravenous 2000 unit / vial Kit; powder, for solution Intravenous 250 unit / vial Kit; powder, for solution Intravenous 3000 unit / vial Kit; powder, for solution Intravenous 500 unit / vial Kit; powder, for solution Intravenous 750 unit / vial Injection, powder, lyophilized, for solution Intravenous 1000 IU Injection, powder, lyophilized, for solution Intravenous 250 IU Injection, powder, lyophilized, for solution Intravenous 500 IU - Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Solid
- Experimental Properties
- Not Available
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Yes
- Actions
- Binding
- General Function
- Important in the maintenance of hemostasis, it promotes adhesion of platelets to the sites of vascular injury by forming a molecular bridge between sub-endothelial collagen matrix and platelet-surface receptor complex GPIb-IX-V. Also acts as a chaperone for coagulation factor VIII, delivering it to the site of injury, stabilizing its heterodimeric structure and protecting it from premature clearance from plasma
- Specific Function
- Collagen binding
- Gene Name
- VWF
- Uniprot ID
- P04275
- Uniprot Name
- von Willebrand factor
- Molecular Weight
- 309261.83 Da
Drug created at June 24, 2016 19:15 / Updated at June 03, 2022 07:24