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General Description:

Elapegademase, identified by CAS number 1709806-75-6, is a notable compound with significant therapeutic applications. Elapegademase is a PEGylated recombinant adenosine deaminase. It can be defined molecularly as a genetically modified bovine adenosine deaminase with a modification in cysteine 74 for serine and with about 13 methoxy polyethylene glycol chains bound via carbonyl group in alanine and lysine residues. Elapegademase is generated in _E. coli_, developed by Leadiant Biosciences and FDA approved on October 5, 2018.

Indications:

This drug is primarily indicated for: Elapegademase is approved for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. This condition was previously treated by the use of bovine pegamedase as part of an enzyme replacement therapy. ADA-SCID is a genetically inherited disorder that is very rare and characterized by a deficiency in the adenosine deaminase enzyme. The patients suffering from this disease often present with a compromised immune system. This condition is characterized by very low levels of white blood cells and immunoglobulin levels which results in severe and recurring infections. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Elapegademase undergoes metabolic processing primarily in: Metabolism studies have not been performed but it is thought to be degraded by proteases to small peptides and individual amino acids. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Elapegademase are crucial for its therapeutic efficacy: Elapegademase is administered intramuscularly and the reported Tmax, Cmax and AUC are approximately 60 hours, 240 mmol.h/L and 33000 hr.mmol/L as reported during a week. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Elapegademase is an important consideration for its dosing schedule: This pharmacokinetic property has not been fully studied. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Elapegademase exhibits a strong affinity for binding with plasma proteins: This pharmacokinetic property is not significant as the main effect is in the blood cells. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Elapegademase from the body primarily occurs through: This pharmacokinetic property has not been fully studied. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Elapegademase is distributed throughout the body with a volume of distribution of: This pharmacokinetic property has not been fully studied. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Elapegademase is a critical factor in determining its safe and effective dosage: This pharmacokinetic property has not been fully studied. It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Elapegademase exerts its therapeutic effects through: In clinical trials, elapegademase was shown to increase adenosine deaminase activity while reducing the concentrations of toxic metabolites which are the hallmark of ADA-SCID. As well, it was shown to improve the total lymphocyte count. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Elapegademase functions by: The ADA-SCID is caused by the presence of mutations in the ADA gene which is responsible for the synthesis of adenosine deaminase. This enzyme is found throughout the body but it is mainly active in lymphocytes. The normal function of adenosine deaminase is to eliminate deoxyadenosine, created when DNA is degraded, by converting it into deoxyinosine. This degradation process is very important as deoxyadenosine is cytotoxic, especially for lymphocytes. Immature lymphocytes are particularly vulnerable as deoxyadenosine kills them before maturation making them unable to produce their immune function. Therefore, based on the causes of ADA-SCID, elapegademase works by supplementing the levels of adenosine deaminase. Being a recombinant and an _E. coli_-produced molecule, the use of this drug eliminates the need to source the enzyme from animals, as it was previously. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Classification:

Elapegademase 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:

Elapegademase is categorized under the following therapeutic classes: Adenosine Deaminase Inhibitors, Adjuvants, Immunologic, Amino Acids, Peptides, and Proteins, Aminohydrolases, Antineoplastic and Immunomodulating Agents, Enzyme Inhibitors, Enzyme Replacement Therapy, Enzymes, Enzymes and Coenzymes, Hydrolases, Nucleoside Deaminases, Pegylated agents, Proteins, Recombinant Adenosine Deaminases, 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 Elapegademase include:

• Molecular Weight: 115000.0
• Molecular Formula: C1797H2795N477O544S12