Eladocagene Exuparvovec
KEBILIDI is a gene therapy medication designed to treat aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic neurological disorder. It works by delivering a functional copy of the DDC gene directly into the brain via intraputaminal infusion, allowing cells to produce the AADC enzyme and subsequently generate dopamine. This treatment is specifically for patients with confirmed AADC deficiency who lack sufficient dopamine production.
KEBILIDI is a gene therapy for AADC deficiency that enables dopamine production by delivering a functional DDC gene directly into the brain.
Key facts at a glance
- ATC
- A16AB26
- RxNorm
- 2698762
- Generic
- Eladocagene Exuparvovec
- Class
- atc-a16
- Brands
- Kebilidi
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 1.4 x 10^11 vg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1.0 x 10^11 vg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 1.2 x 10^11 vg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
7 groupspregnancy
all trimestersavoid use
avoid use during pregnancy due to unknown risks; women of childbearing potential should use effective contraception
regular fetal monitoring if exposure occurs
lactation
breastfeedingnot recommended
breastfeeding not recommended during treatment and for some time after; consult physician
monitor infant for adverse effects
pediatric_age
childrenno adjustment
indicated for pediatric patients with AADC deficiency; administered by qualified neurosurgeon
neurological monitoring, MRI scans, and regular developmental assessments
geriatric
elderlyuse with caution
limited data in elderly populations; assess overall health status and comorbidities
comprehensive geriatric assessment and monitoring for adverse effects
renal_impairment
mild to moderateno specific adjustment
limited renal excretion data; use with caution
monitor renal function
hepatic_impairment
mild to moderateno specific adjustment
limited hepatic metabolism data; use with caution
monitor hepatic function
other
immunocompromiseduse with caution
immunocompromised patients may have increased risk of infections; evaluate immune status
monitor for infections and immune response
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Recombinant AAV vector-based gene therapy
Indication
Treatment of AADC deficiency in pediatric patients
Mechanism of action
AAV vector-mediated gene replacement therapy
Treatment of aromatic L-amino acid decarboxylase (AADC) deficiency, a rare autosomal recessive metabolic disorder characterized by deficient synthesis of dopamine and serotonin.
Delivers a recombinant adeno-associated virus serotype 2 (rAAV2) carrying the DDC gene into the putamen, where cells begin expressing the AADC enzyme and producing dopamine.
Administered as a single intraputaminal infusion procedure. As a gene therapy, effects are intended to be long-lasting. Requires surgical procedure for delivery. Patients should be monitored for neurological improvements over time.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy