Eladocagene Exuparvovec

KEBILIDI 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 KEBILIDI results in AADC enzyme expression and subsequent production of dopamine in the putamen.

• KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on the change from baseline in gross motor milestone achievement at 48 weeks post treatment [see Clinical Studies ( 14 )] .

• KEBILIDI is contraindicated in patients who have not achieved skull maturity assessed by neuroimaging. Skull maturity is needed for stereotactic neurosurgical administration of KEBILIDI.contraindicated

For single-dose intraputaminal infusion only. For single-dose intraputaminal infusion only. Recommended dose: 1.8×10 11 vector genomes (vg). ( 2.2 ) Brain imaging for stereotactic planning and intraoperative navigation should be done prior to the procedure. ( 2.4 ) Post stereotactic registration, mark the entry point on the skull. Surgical access through the skull bone and dura should be performed. ( 2.4 ) Administer a total dose of 1.8×10 11 vg (0.32 mL total volume) delivered as four 0.08 mL (0.45×10 11 vg) infusions (two sites per putamen-anterior and posterior) at a rate of 0.003 mL/minute (0.18 mL/hour) for a total of 27 minutes per site, administered in a single stereotactic surgery using a cannula that is FDA-authorized for intraparenchymal infusion. ( 2.2 , 2.4 ) 2.1 Important Dosing Information Confirm patient has AADC deficiency due to biallelic mutations in the DDC gene. Strictly observe aseptic technique during preparation and administration of KEBILIDI. KEBILIDI should be administered in a medical center which specializes in stereotactic neurosurgery. Administer KEBILIDI only using an FDA-authorized cannula for intraparenchymal infusion (i.e., ClearPoint SmartFlow Neuro Cannula Part Number NGS-NC-01-EE or NGS-NC-02-EE). Use of the syringe (i.e., connecting the syringe to the syringe pump and priming of the cannula) should begin within 6 hours of starting product thaw KEBILIDI is intended to be administered with an infusion pump capable of infusing at a rate of 0.003 mL/min.

Procedural complications: Monitor patients for procedural complications for neurosurgery, including events of respiratory and cardiac arrest after administration of KEBILIDI. ( 5.1 ) Dyskinesia: Monitor patients for dyskinesia after treatment with KEBILIDI. The use of dopamine antagonists can be used to control dyskinesia symptoms. ( 5.2 ) 5.1 Procedural Complications Procedural complications have been reported after neurosurgery required for KEBILIDI administration. These events included respiratory and cardiac arrest which occurred within 24 hours of the neurosurgical procedure and during post-surgical care [see Adverse Reactions (6) ] . KEBILIDI administration has the potential risk for additional procedure related adverse events including cerebrospinal fluid (CSF) leak, intracranial bleeding, neuroinflammation, acute infarction, and infection. Monitor patients for procedure related adverse events with KEBILIDI administration, including continuous cardiorespiratory monitoring during hospitalization. 5.2 Dyskinesia Dyskinesia was reported after administration of KEBILIDI. All events were reported within 3 months of administration and 2 events required hospitalization [see Adverse Reactions (6) ] . Monitor patients for signs and symptoms of dyskinesia after KEBILIDI treatment which may include involuntary movements of face, arm, leg, or entire body. These may present as fidgeting, writhing, wriggling, head bobbing or body swaying. The use of dopamine antagonists may be considered to control dyskinesia symptoms.

Most common adverse reactions (incidence ≥15%) were dyskinesia, pyrexia, hypotension, anemia, salivary hypersecretion, hypokalemia, hypophosphatemia, insomnia, hypomagnesemia, and procedural complications. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact PTC Therapeutics, Inc at toll-free phone 1 866 562 4620 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data described in this section reflects exposure to KEBILIDI in 13 pediatric patients with genetically confirmed AADC deficiency who received a single dose of 1.8×10 11 vg. The median duration of follow-up was 72 weeks (range 23 to 109 weeks) [see Clinical Studies ( 14 )] . The most common adverse reactions (incidence ≥15%) are summarized in Table 3 . Table 3: Adverse Reactions in ≥15% of Patients in Study 1 * Procedural complications included respiratory and cardiac arrest.

8.1 Pregnancy Risk Summary There are no clinical data from the use of KEBILIDI in pregnant women. Animal reproductive and developmental toxicity studies have not been conducted with KEBILIDI. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There is no data on the presence of KEBILIDI in human milk, the effects on the breastfed infant, or the effects on milk production. 8.3 Females and Males of Reproductive Potential Pregnancy Testing Pregnancy status of females with reproductive potential should be verified. Sexually active females of reproductive potential should have a negative pregnancy test before administering KEBILIDI. Contraception There are insufficient exposure data to provide a recommendation concerning duration of contraception following treatment with KEBILIDI. Infertility There is no data on the effects of KEBILIDI on fertility. 8.4 Pediatric Use The safety and effectiveness of KEBILIDI have been established in pediatric patients. The use of KEBILIDI was evaluated in a single-arm, open-label study that enrolled 13 pediatric patients aged 16 months to 10 years who had achieved skull maturity [see Adverse Reactions ( 6 ) and Clinical Studies ( 14 )] .

Metabolism

Biodistribution (within the body) and Vector Shedding (excretion/secretion) KEBILIDI vector DNA levels in various tissues and secretions were determined using a validated quantitative polymerase chain reaction (qPCR) assay. Nonclinical data Biodistribution of eladocagene exuparvovec-tneq was evaluated in rats at Days 7, 30, 90, and 180 after single-dose intraputaminal infusion at dose levels up to 7.5×10 9 vg/animal (21-fold higher than the recommended human dose based on relative brain weight).

1. 1Anxiety133%
2. 2Dyskinesia133%
3. 3Inflammatory Marker Increased133%
4. 4Procedural Headache133%
5. 5Pyrexia133%
6. 6Subdural Haematoma133%

The 1 most recently updated of 1 ClinicalTrials.gov registrations naming Eladocagene Exuparvovec as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Participants
  Active not recruiting · Phase 2 · Interventional · 13 enrolled · PTC Therapeutics
  NCT04903288updated 2026-04-24

Frequently asked questions

How does Eladocagene Exuparvovec work?

KEBILIDI 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 KEBILIDI results in AADC enzyme expression and subsequent production of dopamine in the putamen.

What is Eladocagene Exuparvovec used for?

According to FDA labeling, Eladocagene Exuparvovec carries indications including: KEBILIDI (eladocagene exuparvovec-tneq) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. This indication is approved under accelerated approval based on the change from baseline in gross motor milestone achievement at 48 weeks post treatment [see Clinical Studies ( 14 )] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Eladocagene Exuparvovec?

Eladocagene Exuparvovec is classified as Enzymes, Gene Transduction or Replacement, Increased Dopamine Activity.

What are the brand names for Eladocagene Exuparvovec?

Eladocagene Exuparvovec is marketed under brand names including Kebilidi.

What are the contraindications for Eladocagene Exuparvovec?

Eladocagene Exuparvovec labeling lists contraindications including: KEBILIDI is contraindicated in patients who have not achieved skull maturity assessed by neuroimaging. Skull maturity is needed for stereotactic neurosurgical administration of KEBILIDI.. Always consult the full prescribing information and a clinician.