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Eladocagene Exuparvovec-tneq (Monograph)

Brand name: Kebilidi
Drug class: Gene Therapy

Introduction

Eladocagene exuparvovec-tneq is a recombinant adeno-associated virus (AAV) vector-based gene therapy.

Uses for Eladocagene Exuparvovec-tneq

Eladocagene exuparvovec-tneq has the following uses:

Eladocagene exuparvovec-tneq is indicated for the treatment of adult and pediatric patients with aromatic L amino acid decarboxylase (AADC) deficiency and has been designated an orphan drug by FDA for treatment of this condition.

This indication is approved under accelerated approval based on change from baseline in gross motor milestone achievement at 48 weeks post-treatment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.

Efficacy of eladocagene exuparvovec-tneq was evaluated in an open-label, single arm study in pediatric patients (1.3 to 10.8 years of age) with genetically confirmed, severe AADC deficiency who had achieved skull maturity assessed with neuroimaging. Patients received a single total dose of 1.8×1011 vg of eladocagene exuparvovec-tneq given as four intraputaminal infusions in a single stereotactic neurosurgical procedure. Gross motor milestone achievement at week 48 was assessed as the primary efficacy outcome measure. The comparison group was an external untreated natural history cohort of 43 pediatric patients with severe AADC deficiency. In the 12 patients treated with eladocagene exuparvovec-tneq, 67% achieved a new gross motor milestone at week 48 compared to none of the 43 untreated patients from the historical control.

Eladocagene Exuparvovec-tneq Dosage and Administration

General

Eladocagene exuparvovec-tneq is available in the following dosage form(s) and strength(s):

Dosage

It is essential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

Dosage and Administration in Adults and Pediatric Patients

Cautions for Eladocagene Exuparvovec-tneq

Contraindications

Warnings/Precautions

Procedural Complications

Procedural complications have been reported after neurosurgery required for eladocagene exuparvovec-tneq administration. These events included respiratory and cardiac arrest which occurred within 24 hours of the neurosurgical procedure and during post-surgical care. Eladocagene exuparvovec-tneq 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 eladocagene exuparvovec-tneq administration, including continuous cardiorespiratory monitoring during hospitalization.

Dyskinesia

Dyskinesia was reported after administration of eladocagene exuparvovec-tneq. All events were reported within 3 months of administration and 2 events required hospitalization.

Monitor patients for signs and symptoms of dyskinesia after eladocagene exuparvovec-tneq treatment, which may include involuntary movements of the 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.

Specific Populations

Pregnancy

There are no clinical data from the use of eladocagene exuparvovec-tneq in pregnant women. Animal reproductive and developmental toxicity studies have not been conducted with eladocagene exuparvovec-tneq.

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.

Lactation

There is no data on the presence of eladocagene exuparvovec-tneq in human milk, the effects on the breastfed infant, or the effects on milk production.

Females and Males of Reproductive Potential

Pregnancy status of females with reproductive potential should be verified. Sexually active females of reproductive potential should have a negative pregnancy test before administering eladocagene exuparvovec-tneq.

There are insufficient exposure data to provide a recommendation concerning duration of contraception following treatment with eladocagene exuparvovec-tneq.

There is no data on the effects of eladocagene exuparvovec-tneq on fertility.

Pediatric Use

The safety and effectiveness of eladocagene exuparvovec-tneq have been established in pediatric patients. The use of eladocagene exuparvovec-tneq was evaluated in a single-arm, open-label study that enrolled 13 pediatric patients 16 months to 10 years of age who had achieved skull maturity.

The safety and effectiveness of eladocagene exuparvovec-tneq have not been studied in pediatric patients younger than 16 months of age.

Geriatric Use

Eladocagene exuparvovec-tneq has not been studied in patients 65 years of age and older.

Common Adverse Effects

Most common adverse reactions (incidence ≥15%) were dyskinesia, pyrexia, hypotension, anemia, salivary hypersecretion, hypokalemia, hypophosphatemia, insomnia, hypomagnesemia, and procedural complications.

Drug Interactions

Specific Drugs

It is essential that the manufacturer's labeling be consulted for more detailed information on interactions with this drug, including possible dosage adjustments. Interaction highlights:

Please see product labeling for drug interaction information.

Actions

Mechanism of Action

Eladocagene exuparvovec-tneq is a recombinant adeno-associated virus serotype 2 (rAAV2) based gene therapy designed to deliver a copy of the DDC gene which encodes the aromatic L amino acid decarboxylase (AADC) enzyme. Intraputaminal infusion of eladocagene exuparvovec-tneq results in AADC enzyme expression and subsequent production of dopamine in the putamen.

Advice to Patients

Additional Information

AHFSfirstRelease. For additional information until a more detailed monograph is developed and published, the manufacturer's labeling should be consulted. It is essential that the manufacturer's labeling be consulted for more detailed information on usual uses, dosage and administration, cautions, precautions, contraindications, potential drug interactions, laboratory test interferences, and acute toxicity.

Preparations

Excipients in commercially available drug preparations may have clinically important effects in some individuals; consult specific product labeling for details.

Please refer to the ASHP Drug Shortages Resource Center for information on shortages of one or more of these preparations.

Eladocagene Exuparvovec-tneq

Routes

Dosage Forms

Strengths

Brand Names

Manufacturer

Local

Suspension, for Intraputaminal Infusion

nominally 5.6 x 1011 vector genomes (vg)/mL

Kebilidi

PTC Therapeutics

AHFS DI Essentials™. © Copyright 2025, Selected Revisions November 27, 2024. American Society of Health-System Pharmacists, Inc., 4500 East-West Highway, Suite 900, Bethesda, Maryland 20814.

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