Version July 2025
Dosage guidance:
Safety: Administer during a stereotactic neurosurgical procedure.
Clinical considerations: Confirm biallelic mutations in DDC gene to confirm aromatic L-amino acid decarboxylase (AADC) deficiency prior to administering.
Aromatic L-amino acid decarboxylase (AADC) deficiency, treatment:
Intraputaminal: 1.8 × 1011 vg (0.32 mL) administered as 4 intraputaminal infusions (0.45 × 1011 vg [0.08 mL] per infusion; 2 infusions in the anterior putamen and 2 infusions in the posterior putamen) in a single stereotactic neurosurgical procedure. Refer to manufacturer's labeling for additional details.
There are no dosage adjustments provided in the manufacturer's labeling.
There are no dosage adjustments provided in the manufacturer's labeling.
Refer to adult dosing.
(For additional information see "Eladocagene exuparvovec: Pediatric drug information")
Dosage guidance:
Safety: Administer during a neurosurgical procedure in a center that specializes in stereotactic neurosurgery.
Clinical considerations: Confirm aromatic L-amino acid decarboxylase (AADC) deficiency due to biallelic mutations in DDC gene prior to administering.
Aromatic L-amino acid decarboxylase (AADC) deficiency:
Children ≥16 months and Adolescents: Intraputaminal: 1.8 × 1011 vector genomes (vg) (0.32 mL) administered as 4 intraputaminal infusions (0.08 mL each; 2 in anterior putamen and 2 in posterior putamen) during a single stereotactic neurosurgical procedure using an FDA-authorized cannula for intraparenchymal infusion.
There are no dosage adjustments provided in the manufacturer's labeling.
There are no dosage adjustments provided in the manufacturer's labeling.
The following adverse drug reactions are derived from product labeling unless otherwise specified. Adverse reactions are reported in children.
>10%:
Cardiovascular: Hypotension
Endocrine & metabolic: Hypokalemia, hypomagnesemia, hypophosphatemia
Gastrointestinal: Sialorrhea
Hematologic & oncologic: Anemia
Immunologic: Antibody development (including neutralizing)
Nervous system: Insomnia
Neuromuscular & skeletal: Dyskinesia
Miscellaneous: Fever
1% to 10%: Ophthalmic: Oculogyric crisis
Patients who have not achieved skull maturity assessed by neuroimaging; skull maturity is necessary for stereotactic neurosurgical administration.
Concerns related to adverse effects:
• Dyskinesias: Dyskinesias, including those resulting in hospitalization, have been reported; dyskinesias typically occur within 3 months of administration. Dyskinesias, including involuntary movements of the arms, legs, face, or entire body, may be characterized by body swaying, fidgeting, head bobbing, or writhing. Consider utilizing dopamine antagonists to control dyskinesia symptoms.
• Procedural complications: Procedural complications, including respiratory and cardiac arrest, have been reported; complications may occur within 24 hours of the neurosurgical procedure as well as during postsurgical care. Other procedural complications include acute infarction, cerebrospinal fluid leak, infection, intracranial bleeding, and neuroinflammation.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Suspension, Injection [preservative free]:
Kebilidi: Eladocagene exuparvovec-tneq 28 billion VG/0.5 mL (0.5 mL)
No
Suspension (Kebilidi Injection)
280000000000VG/0.5ML (per 0.5 mL): $4,740,000.00
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Intraputaminal: Administer in a medical center specializing in stereotactic neurosurgery using an FDA-authorized cannula for intraparenchymal infusion (eg, ClearPoint SmartFlow Neuro Cannula Part Number NGS-NC-01-EE or NGS-NC-02-EE) and an infusion pump capable of infusing at a rate of 0.003 mL/minute. Connect syringe to syringe pump and prime cannula within 6 hours of starting product thaw. Administer each infusion (eg, at each putaminal target site) over 27 minutes at a rate of 0.003 mL/minute. Refer to manufacturer's labeling for additional details.
Intraputaminal: Administer in a medical center specializing in stereotactic neurosurgery; refer to manufacturer's labeling for additional details. Recommended dose should be divided into 4 different infusions of 0.08 mL (0.45 × 1011 vector genomes [vg]) each; administer 2 infusions into anterior putamen and 2 infusions into posterior putamen. Administer each infusion consecutively, over 27 minutes per site, at a rate of 0.003 mL/minute using a syringe pump and an FDA-authorized cannula for intraparenchymal infusion (ie, ClearPoint SmartFlow Neuro Cannula Part Number NGS-NC-01-EE or 122 NGS-NC-02-EE); total infusion time is ~4 hours.
Aromatic L-amino acid decarboxylase deficiency: Treatment of aromatic L-amino acid decarboxylase (AADC) deficiency in adults and pediatric patients.
None known.
There are no known significant interactions.
Evaluate pregnancy status prior to use; sexually active patients should have a negative pregnancy test confirmed prior to starting treatment.
Data are insufficient to recommend the duration of contraception required following treatment with eladocagene exuparvovec.
Animal reproduction studies have not been conducted.
It is not known if eladocagene exuparvovec is present in breast milk.
Continuous cardiorespiratory monitoring (throughout the entire hospitalization for neurosurgical administration).
Eladocagene exuparvovec is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the DDC gene, which encodes the AADC enzyme. Intraputaminal infusion of eladocagene exuparvovec results in AADC enzyme expression and subsequent production of dopamine in the putamen.
