Simoctocog Alfa, Recombinant B-domain deleted (BDD) rFVIII

Specification

• Cat#: THP-0092
• Product Name: Simoctocog Alfa, Recombinant B-domain deleted (BDD) rFVIII
• Description: The product is a recombinant B-domain deleted (BDD) rFVIII produced in HEK293F cells. The harvested product is concentrated and purified by a series of chromatography steps. The product is a glycoprotein consisting of 1440 amino acids with an approximate molecular mass of 170 kDa, comprising the FVIII domains A1-A2 + A3-C1-C2 whereas the B-domain, present in the full-length plasma-derived FVIII, has been deleted and replaced by a 16 amino acid linker.
• Sequences: Not Available
• Species: Human
• Molecular Weight: 170000.0 Da (Approximate, B-Domain deleted)
• Source: HEK293 Cells
• Purity: >99% by SDS-Page and HPLC analysis
• Cas No: 1219013-68-9
• Formula: Not Available
• Endotoxin Level: <0.001 EU per 1 μg by the LAL method
• Biological Activity: 2000IU/2.5mL

Pharmaceutical Information

• Pharmaceutical Application: Simoctocog Alfa
• Applications: Indicated for the treatment and prophylaxis of bleeding in patients of all ages with haemophilia A (congenital factor VIII deficiency).
• Examples of Clinical Use: Haemophilia A (congenital factor VIII deficiency)
• Pharmacodynamics: The product is an antihemorrhagic agent that displays comparable hemostatic efficacy as the endogenous coagulation factor VIII. Following administration of the product, an increase plasma levels of factor VIII is observed to temporarily correct factor VIII deficiency and bleeding tendencies.
• Mechanism of action: Haemophilia A is a sex-linked hereditary disorder of blood coagulation due to decreased levels of factor VIII:C. The disorder is associated with episodes of recurrent bleeding and profuse bleeding into joints, muscles or internal organs, either spontaneously or as results of accidental or surgical trauma.
• Affected organisms: Not Available
• Targets: Target 1. von Willebrand factor

Related Reading

Background of Simoctocog Alfa

Simoctocog Alfa, also known as Nuwiq, is a recombinant Factor VIII (FVIII) derived from human cells. It's developed to treat patients diagnosed with Hemophilia A, an X-linked genetic disorder characterized by the deficiency or dysfunction of FVIII. The discovery of Simoctocog Alfa can be traced back to the combined efforts of early pioneers in genetic science and biotechnology, striving to address the complications related to the treatment of Hemophilia A.
Simoctocog Alfa’s gene locus is situated on the X chromosome, specifically Xq28. As for its protein structure, it contains the same amino acid sequence as human FVIII, consisting of six domains encoded by different exons. The high-efficiency expression construct allows for the human post-translational modifications to occur, making Simoctocog Alfa remarkably similar to the natural FVIII factor.

Simoctocog Alfa Function

Simoctocog Alfa functions similar to endogenous Factor VIII by playing a crucial role in the clotting cascade, which is responsible for blood coagulation. Replacing the missing coagulation Factor VIII in patients with hemophilia A, it promotes blood clotting and effectively controls and prevents bleeding.

Simoctocog Alfa-Related Signaling Pathways

The functionality of Simoctocog Alfa is highly dependent on several signaling pathways. The most significant is the coagulation cascade—a series of reactions designed to form a clot and prevent excessive bleeding. In this sequence, FVIII binds to Von Willebrand factor (VWF), which is found in the blood, and is carried to the site of injury. Here, it interacts with activated Factor IX (FIXa) to form the tenase complex, responsible for the conversion of Factor X to its activated version (FXa), thereby facilitating the production of a fibrin clot.

Simoctocog Alfa and Diseases

As mentioned, Simoctocog Alfa is integral in managing Hemophilia A, a disorder marked by a lack of functional FVIII, resulting in difficulties with clotting. Simoctocog Alfa serves as a substitute for the missing coagulation factor and allows for effective clot formation. The application of Simoctocog Alfa can help control spontaneous bleeding and reduce the frequency of bleeding episodes. In addition, this pharmaceutical intervention plays a significant role in surgical and post-operative management in individuals with Hemophilia A.

Application of Simoctocog Alfa in Medicine

Given its efficacy in replacing the FVIII factor, Simoctocog Alfa has become one of the primary treatments for patients with Hemophilia A. It's administered via intravenous injection, either prophylactically to prevent bleedings or on-demand to treat acute bleeding episodes.

List of Drug Candidates Related to Simoctocog Alfa

Simoctocog Alfa, commercially known as Nuwiq, comes in several forms with distinct international non-proprietary names, including Moroctocog Alfa (Refacto), Turoctocog Alfa (Novoeight), and the long-acting variant Efmoroctocog Alfa (Elocta). These drug candidates share the common functionality of providing FVIII replacement therapy.