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ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Enzymes
eladocagene exuparvovec
A16AB26
Forms & Strengths
- Suspension for infusion: 2 × 10^11 vector genomes (vg)/mL in a 0.5 mL extractable volume
Adult Dosing
- Not applicable (indicated exclusively for pediatric patients)
Pediatric Dosing
- Aromatic L-amino acid decarboxylase (AADC) deficiency: 1.8 × 10^11 vg total dose administered via stereotactic neurosurgical infusion into the bilateral putamen divided into four trajectories (two per hemisphere)
Indications
- Treatment of patients aged 18 months and older with a clinical, molecular, and genetic diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency
Mechanism of Action
- Recombinant adeno-associated virus serotype 2 (AAV2) vector containing the human DDC gene, designed to deliver a functional DDC gene directly to putaminal neurons to restore AADC enzyme activity and dopamine synthesis
Contraindications
- Previous hypersensitivity to the active substance or any of the excipients
- Active systemic infection
- Severe intracranial structural abnormalities precluding the surgical procedure
Adverse Reactions
- Dyskinesia
- Insomnia
- Pyrexia
- Anxiety
- Hypokinesia
- Procedural complications related to stereotactic neurosurgery (e.g., intracranial hemorrhage, cerebrospinal fluid leak)
Drug Interactions
- Formal drug-drug interaction studies have not been performed
- Use with caution in patients receiving agents affecting central nervous system neurotransmitters or monoamine pathways
Curated Content: Needs Vetting Before Put to Clinical Use