Section 12 · Monograph
Eladocagene exuparvovec
Reviewed Nov 2026FDA label
KebilidiAAV gene therapy
- Forms
- Intraputaminal infusion suspension (AAV2-hAADC gene therapy)
- Dose
- AADC deficiency: one-time stereotactic neurosurgical infusion into the putamen at a specialized center
- MOA
- Delivers a functional DDC (AADC) gene to putaminal neurons
Indications
- Aromatic L-amino acid decarboxylase (AADC) deficiency in adult and pediatric patientsFDA-labeled
Mechanism of Action
- Recombinant AAV2 vector carrying the human DDC (AADC) gene, restoring dopamine synthesis in putaminal neurons
Full Dosing
Adult / usual
- One-time intraputaminal infusion delivered by stereotactic neurosurgery at a specialized center; dose and volume per the current prescribing information
- Repeat treatment is considered investigational
- Immunosuppression and perioperative management per label
Contraindications
- Patients who have not achieved skull maturity as assessed by neuroimaging
Drug Interactions
- No formal drug interaction studies; coordinate with the patient's dopaminergic and anesthetic regimens
Adverse Effects
- Dyskinesia (very common after treatment)
- Pyrexia, hypotension, anemia
- Procedure-related risks (intracranial hemorrhage, device complications, infection)
- Salivary hypersecretion, insomnia, hypokalemia
Monitoring Parameters
- Neurologic exam, dyskinesia and motor milestones
- Postoperative MRI/CT and neurosurgical follow-up
- Long-term safety follow-up
Pregnancy & Lactation
- Not applicable to pregnancy in the pediatric population; limited data