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General Description:

Efgartigimod alfa, identified by CAS number 1821402-21-4, is a notable compound with significant therapeutic applications. Myasthenia gravis (MG) is an autoimmune disorder characterized by significant muscle weakness - particularly in the eye, throat, and extremities - caused by autoantibodies attacking the neuromuscular junction. The production of IgG autoantibodies against acetylcholine receptors (AChRs) is one of the more common pathophysiological mechanisms behind MG, and results in the destruction of these receptors and a reduction in electrical nerve impulses. Efgartigimod alfa is a first-in-class antagonist of the neonatal Fc receptor (FcRn) used in the treatment of myasthenia gravis (MG). IgG antibodies, including the autoantibodies responsible for MG symptoms, can be 'recycled', a process which significantly extends their half-life, by evading lysosomal degradation via binding with FcRn. By antagonizing this interaction, efgartimod alfa prevents this recycling phase and thus decreases the half-life of IgG, effectively lowering circulating levels of IgG autoantibodies against AChRs. Efgartimod alfa was granted FDA approval on December 17, 2021 and European Commission approval on August 11, 2022.

Indications:

This drug is primarily indicated for: Efgartigimod alfa is indicated for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor antibody-positive. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Efgartigimod alfa undergoes metabolic processing primarily in: As with other therapeutic proteins, efgartigimod alfa is likely metabolized to smaller peptides and amino acids via proteolytic enzymes. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Half-life:

The half-life of Efgartigimod alfa is an important consideration for its dosing schedule: The terminal elimination half-life of efgartigimod alfa ranges from 80 to 120 hours. This determines the duration of action and helps in formulating effective dosing regimens.

Route of Elimination:

The elimination of Efgartigimod alfa from the body primarily occurs through: After a single intravenous dose of efgartigimod alfa 10 mg/kg in healthy subjects, less than 0.1% of the administered dose was recovered in the urine. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Efgartigimod alfa is distributed throughout the body with a volume of distribution of: The volume of distribution of efgartigimod alfa ranges from 15 to 20 liters. This metric indicates how extensively the drug permeates into body tissues.

Pharmacodynamics:

Efgartigimod alfa exerts its therapeutic effects through: Efgartigimod alfa exerts its pharmacologic effect by reducing circulating levels of the autoantibody responsible for myasthenia gravis symptoms. It is administered as a once-weekly intravenous infusion, given for 4 weeks per treatment cycle, with the option to initiate additional treatment cycles as clinically indicated after at least 50 days have passed following the previous cycle. Because efgartigimod alfa reduces circulating IgG levels, patients undergoing therapy may be at greater risk of infection due to a depressed immune response. It should not be initiated in patients with an active infection, and consideration should be given to holding therapy in patients who develop a serious infection during a treatment cycle. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Efgartigimod alfa functions by: Myasthenia gravis (MG) is an autoimmune disorder affecting the neuromuscular junction of the skeletal muscles. While the pathophysiologic mechanisms of MG differ depending on the subtype in question, all forms involve the production of IgG autoantibodies to some endogenous protein. One of the most commonly implicated proteins against which autoantibodies are produced are acetylcholine receptors (AChRs), which undergo degradation via the membrane attack complex (MAC) secondary to their interaction with AChR-specific autoantibodies. The destruction of AChRs prevents regular transmission of electrical impulses across the neuromuscular junction, which ultimately leads to the characteristic muscular weakness - especially of the eyes, throat, and extremities - observed in patients with MG. Immunoglobulin G, as opposed to other immunoglobulins, undergoes a recycling phase in the vascular endothelium that dramatically extends its half-life. In the case of pathogenic IgGs causing MG, this may facilitate an increased ability to impair neuromuscular transmission. This recycling involves IgG binding to the neonatal Fc receptor (FcRn), which rescues IgG from lysosomal degradation. Efgartigimod alfa is a human IgG1 antibody fragment that binds to FcRn, thus preventing IgG recycling and subsequently reducing the amount of circulating IgG, including the autoantibodies responsible for MG. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Classification:

Efgartigimod 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.

Categories:

Efgartigimod alfa is categorized under the following therapeutic classes: Antibodies, Globulins, Human Immunoglobulin G, Immunoglobulin Fc Fragments, Immunoproteins. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.

Experimental Properties:

Further physical and chemical characteristics of Efgartigimod alfa include:

• Molecular Weight: 54000.0