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General Description:

Vonicog alfa, identified by CAS number 109319-16-6, is a notable compound with significant therapeutic applications. Vonicog alfa is a recombinant von Willebrand factor manufactured by Baxalta. It was FDA approved in December 2015. The gen of von Willebrand factor was first cloned in 1985 by Stuart Orkin and David Ginsburg. By the EMA, vonicog alfa is still under clinical analysis.

Indications:

This drug is primarily indicated for: Vonicog alfa is indicated for the on-demand treatment and control of bleeding episodes in adults previously diagnosed with von Willebrand disease. Vonicog alfa contains only the vWF and thus, its administration offers the flexibility to administer the coagulation factor VIII if needed. The von Willebrand disease is an inherited disorder characterized by the deficiency or misfunction of the von Willebrand factor (vWF). Due to this deficiency, the blood cannot clot properly and the patients that present this disease are prone to prolonged or excessive bleeding. There are three types of this disease, and type 3 is an autosomal recessive inherited disorder marked by very low or absent levels of vWF. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Vonicog alfa undergoes metabolic processing primarily in: The endogenous regulator of the vWF is ADAMTS13. The interaction between these two proteins results in the proteolysis of vWF. This proteolysis occurs primarily in the cleavage site at the domain A2 which is a target domain for ADAMTS13. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Vonicog alfa are crucial for its therapeutic efficacy: In pharmacokinetic studies, the Cmax, AUC and mean residence time of vonicog alfa are reported to be 90.7 mcg, 1877 h.U/dL and 29.8 hours, respectively. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Vonicog alfa is an important consideration for its dosing schedule: Clinical trials have shown a terminal half-life of 21.9 hours for vonicog alfa which can be modified by the coadministration of the factor VIII. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Vonicog alfa exhibits a strong affinity for binding with plasma proteins: Vonicog alfa presents a very high plasma protein binding as its main function is performed in the blood. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Vonicog alfa from the body primarily occurs through: The majority of vonicog alfa is targeted to the liver and spleen which indicates an active regulatory elimination mechanism. It seems to be uptaken mainly by macrophages. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Vonicog alfa is distributed throughout the body with a volume of distribution of: In phase 3 clinical trials, the volume of distribution of vonicog alfa is reported to be of 0.80 L. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Vonicog alfa is a critical factor in determining its safe and effective dosage: In phase 3 clinical trials, the clearance rate of vonicog alfa is reported to be of 0.29 dL/kg/h. It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Vonicog alfa exerts its therapeutic effects through: Vonicog alfa does not present a risk of transmission of blood-borne pathogens as it is a recombinant, plasma- and albumin-free protein. The two first clinical trials showed an effective reduction of bleeding episodes in different sites of the body. This effect was observed even after the first infusion in 81% of the cases. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Vonicog alfa functions by: Von Willebrand factor (vWF) is an important piece of the blood coagulation cascade and it acts by stabilizing the coagulation factor VIII. It also binds to collagen and platelets in blood vessel walls which helps with the formation of a platelet plug during clotting process. The presence of different mutations in vWF causes a common bleeding disorder named von Willebrand disease. The bleeding episodes were usually treated with plasma-derived vWF/factor VIII concentrates which required high doses and long treatment duration. The use of vonicog alfa allows a separate administration of vWF without the presence of the Factor VIII. This separate dosing allows the personalization of the dosage according to the patient's needs. On the same note, the plasma-derived vWF concentrates variably lack ULM due to an in vivo exposure to plasma ADAMTS13 and granulocyte elastases. Vonicog alfa can be delivered as an unaltered vWF and it functions as a replacement of vWF. Thus, it mediates platelet adhesion and aggregation as well as stabilization of the procoagulant factor VIII. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Classification:

Vonicog alfa 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.

Experimental Properties:

Further physical and chemical characteristics of Vonicog alfa include:

• Molecular Weight: 20000000.0