Eladocagene exuparvovec API Manufacturers

General Description:

Eladocagene exuparvovec, identified by CAS number 2098615-91-7, is a notable compound with significant therapeutic applications. Eladocagene exuparvovec is a recombinant adeno-associated virus-2 (AAV2)-based gene therapy that expresses human aromatic L-amino acid decarboxylase (AADC), and it is used to treat AADC deficiency, a fatal and rare genetic disorder that causes severe disability in pediatric patients. Patients with AADC have mutations in the dopa decarboxylase (DDC) gene that encodes the AADC enzyme. By promoting the expression of AADC, eladocagene exuparvovec leads to the development of motor function in patients with AADC deficiency. Eladocagene exuparvovec is infused directly into the brain (putamen) by bilateral intraputaminal infusion in one surgical session. Eladocagene exuparvovec received EMA approval in July 2022. Before the approval of this gene therapy, the treatment options for patients with AADC deficiency were limited to attempts to increase monoamine neurotransmitter production, decrease neurotransmitter catabolism via monoamine oxidase (MAO) inhibition and address symptoms.

Indications:

This drug is primarily indicated for: Eladocagene exuparvovec is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.

Metabolism:

Eladocagene exuparvovec undergoes metabolic processing primarily in: As a gene therapy product, eladocagene exuparvovec is expected to be metabolized by nucleases throughout the body. This metabolic pathway ensures efficient processing of the drug, helping to minimize potential toxicity and side effects.

Absorption:

The absorption characteristics of Eladocagene exuparvovec are crucial for its therapeutic efficacy: Pharmacokinetic studies with eladocagene exuparvovec have not been performed. Eladocagene exuparvovec is infused directly into the brain and has not been shown to distribute outside the CNS. The drug's ability to rapidly penetrate into cells ensures quick onset of action.

Half-life:

The half-life of Eladocagene exuparvovec is an important consideration for its dosing schedule: Not available. This determines the duration of action and helps in formulating effective dosing regimens.

Protein Binding:

Eladocagene exuparvovec exhibits a strong affinity for binding with plasma proteins: Not available. This property plays a key role in the drug's pharmacokinetics and distribution within the body.

Route of Elimination:

The elimination of Eladocagene exuparvovec from the body primarily occurs through: Not available. Understanding this pathway is essential for assessing potential drug accumulation and toxicity risks.

Volume of Distribution:

Eladocagene exuparvovec is distributed throughout the body with a volume of distribution of: The biodistribution of eladocagene exuparvovec was evaluated using a real-time polymerase chain reaction assay. Eladocagene exuparvovec was not detected in blood and urine at baseline or 12 months after treatment. This metric indicates how extensively the drug permeates into body tissues.

Clearance:

The clearance rate of Eladocagene exuparvovec is a critical factor in determining its safe and effective dosage: Not available. It reflects the efficiency with which the drug is removed from the systemic circulation.

Pharmacodynamics:

Eladocagene exuparvovec exerts its therapeutic effects through: The success and stability of aromatic L-amino acid decarboxylase (AADC) gene transduction achieved with eladocagene exuparvovec was evaluated by measuring L-6- fluoro-3, 4-dihydroxyphenylalanine (¹⁸F-DOPA) uptake. An increase in ¹⁸F-DOPA uptake was evident as early as 6 months after treatment, and was sustained for at least 5 years. The use of eladocagene exuparvovec led to motor milestones acquisition as early as 12 months after administration. In patients treated with eladocagene exuparvovec, dyskinesia or insomnia may occur or worsen 1 month after administration. Complications of eladocagene exuparvovec treatment, such as leakage of the fluid surrounding the brain, meningitis, or encephalitis, should be monitored. The drug's ability to modulate various physiological processes underscores its efficacy in treating specific conditions.

Mechanism of Action:

Eladocagene exuparvovec functions by: Eladocagene exuparvovec is a recombinant adeno-associated virus-2 (AAV2) vector that expresses human aromatic L-amino acid decarboxylase (AADC). It is used to treat patients with AADC deficiency, an autosomal recessive condition caused by mutations in the dopa decarboxylase (DDC) gene. The DDC gene encodes the AADC enzyme, which converts L-3,4-dihydroxyphenylalanine (L-DOPA) to dopamine and 5-hydroxytryptophan to serotonin. However, the mutations present in patients with AADC deficiency lead to the reduction or absence of AADC enzyme activity, causing a depletion in neurotransmitter levels and a failure to achieve developmental milestones. Eladocagene exuparvovec is administered to the putamen of patients with AADC deficiency (direct brain infusion), where it drives the production of the AADC enzyme and increases dopamine levels. Consequently, the use of eladocagene exuparvovec improves the development of motor function in treated patients with AADC deficiency. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.

Toxicity:

Categories:

Eladocagene exuparvovec is categorized under the following therapeutic classes: Adeno-associated Viral Vector Therapies, Genetic Therapy. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.