Rurioctocog alfa pegol API Manufacturers

General Description:

Rurioctocog alfa pegol, identified by CAS number 1417412-83-9, is a notable compound with significant therapeutic applications. Rurioctocog alfa pegol is a pegylated recombinant human coagulation factor VIII or antihemophilic factor. Factor VIII is an essential protein involved in normal blood clotting; thus, a deficient level of functional factor VIII is associated with an elevated risk for excessive bleeding caused by spontaneous or secondary events like trauma or surgery. Hemophilia A is the most common inherited bleeding disorder leading to deficiency of factor VIII, which is caused by defects in the F8C gene that encodes coagulation factor VIII. Bleeding in joints is a common manifestation of hemophilia A, and bleeding episodes can be severe and life-threatening like intracranial hemorrhage. Rurioctocog alfa pegol aims to restore functional levels of factor VIII in patients with hemophilia A to manage and prevent bleeding episodes. It was first approved by the European Commission in January 2018. Rurioctocog alfa pegol is a covalent conjugate of , which is a recombinant factor VIII produced by recombinant DNA technology from a Chinese hamster ovary cell line. The presence of the polyethylene glycol (PEG) moiety increases the plasma half-life of the drug, thereby increasing the drug's duration of action.

Indications:

This drug is primarily indicated for: Rurioctocog alfa pegol is indicated for the treatment and prophylaxis of bleeding in patients 12 years and above with hemophilia A (congenital factor VIII deficiency). Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Rurioctocog alfa pegol undergoes metabolic processing primarily in: There is limited information regarding the metabolism of rurioctocog alfa pegol; however, it is expected to undergo catabolism just like endogenous coagulation factor VIII. Metabolism pathways and CYP involvement are not known to exist regarding coagulation factors. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Rurioctocog alfa pegol are crucial for its therapeutic efficacy: Following intravenous administration of rurioctocog alfa pegol, the mean ± SD C_max was 117 ± 28 IU/dL in adolescents and 145 ± 29 IU/dL in adults. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Rurioctocog alfa pegol is an important consideration for its dosing schedule: Following intravenous administration of rurioctocog alfa pegol, the mean ± SD terminal half-life was 13.80 ± 4.01 hours in adolescents and 15.01 ± 3.89 hours in adults. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Rurioctocog alfa pegol exhibits a strong affinity for binding with plasma proteins: Once rurioctocog alfa pegol enters the circulation, it binds to von Willebrand factor, just like how an endogenous factor VIII would. There is limited information on the binding profile of rurioctocog alfa pegol to plasma proteins other than von Willebrand factor. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Rurioctocog alfa pegol from the body primarily occurs through: There is limited information regarding the route of elimination of rurioctocog alfa pegol. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Rurioctocog alfa pegol is distributed throughout the body with a volume of distribution of: Following intravenous administration, the mean ± SD volume of distribution at steady-state was 0.54 ± 0.22 dL/kg in adolescents and 0.40 ± 0.09 dL/kg in adults. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Rurioctocog alfa pegol is a critical factor in determining its safe and effective dosage: Following intravenous administration of rurioctocog alfa pegol, the mean ± SD clearance was 2.58 ± 0.84 mL/(kg·h) in adolescents and 2.16 ± 0.75 mL/(kg·h) in adults. It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Rurioctocog alfa pegol exerts its therapeutic effects through: Rurioctocog alfa pegol is a recombinant coagulation factor that promotes blood clotting. In clinical trials, personalized administration of rurioctocog alfa pegol based on each patient's clinical profile led to increased plasma levels of factor VIII and reduced bleeding episodes at six and 12 months of treatment. Rurioctocog alfa pegol has a long duration of action compared to : it has an extended half-life of 1.4 to 1.5-fold compared to octocog alfa in adolescents and adults. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Rurioctocog alfa pegol functions by: Factor VIII plays an essential role in the intrinsic pathway of the blood coagulation cascade: as a cofactor for activated factor IX (FIXa), factor VIII significantly facilitates the conversion of FIXa-mediated transformation of inactive factor X (FX) to its active form (FXa). FXa then promotes the conversion of prothrombin into thrombin, a key serine protease of the coagulation cascade. Proper blood clotting cannot be achieved with the deficient levels of factor VIII, leading to increased excessive bleeding spontaneously or as a result of accidental or surgical trauma. Hemophilia A is an X-chromosomal linked hereditary disorder of blood coagulation due to defects in the F8C gene that encodes coagulation factor VIII, leading to decreased production of functional factor VIII. Rurioctocog alfa pegol is a pegylated recombinant human factor VIII that aims to restore the plasma levels and hemostatic activity of functional factor VIII. It can be used for both prophylactic or on-demand treatment of bleeding episodes associated with hemophilia A. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Classification:

Rurioctocog alfa pegol belongs to the None, classified under the direct parent group Peptides. This compound is a part of the Organic Compounds, falling under the Organic Acids superclass, and categorized within the Carboxylic Acids and Derivatives class, specifically within the Amino Acids, Peptides, and Analogues subclass.

Categories:

Rurioctocog alfa pegol is categorized under the following therapeutic classes: Amino Acids, Peptides, and Proteins, Biological Factors, Blood Coagulation Factors, Blood Proteins, Coagulants, Factor VIII, Hematologic Agents, Hemostatics, Pegylated agents, Protein Precursors, Proteins, Recombinant Proteins. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.

Experimental Properties:

Further physical and chemical characteristics of Rurioctocog alfa pegol include:

• Molecular Weight: 269812.0
• Molecular Formula: C12257H17863N3220O3552S83