Eladocagene Exuparvovec-tneq

Eladocagene Exuparvovec is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency in adults and pediatric patients.

• Eladocagene Exuparvovec is available under the following different brand names: Kebilidi, Eladocagene Exuparvovec-tneq.

Common side effects of Eladocagene Exuparvovec include:

• uncontrolled, involuntary muscle movements (dyskinesia)
• fever
• hypotension 
• anemia
• salivary hypersecretion 
• hypokalemia
• hypophosphatemia 
• insomnia
• hypomagnesemia
• procedural complications

Serious side effects of Eladocagene Exuparvovec include:

• procedural complications
• uncontrolled, involuntary muscle movements (dyskinesia)

Rare side effects of Eladocagene Exuparvovec include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult and pediatric dosage

Suspension for intraputaminal infusion

• 2.8x10^11 vector genomes (vg)/0.5 mL in single-dose vial
• 0.5 mL extractable volume per 2 mL vial

Aromatic L-amino acid decarboxylase (AADC) deficiency

Adult dosage

• Total dose of 1.8 x10^11 vector genomes (vg) (in 0.32 mL) given in 4 intraputaminal infusions of 0.45 x 10^11 vg (0.08 mL) per dose in single stereotactic neurosurgical procedure
• Administer 2 infusions in anterior putamen and 2 infusions in posterior putamen

Pediatric dosage

• Children younger than 16 months: Safety and efficacy not established
• Children aged 16 months and older
• Total dose of 1.8 x10^11 vector genomes (vg) (in 0.32 mL) given in 4 intraputaminal infusions of 0.45 x 10^11 vg (0.08 mL) per dose in a single stereotactic neurosurgical procedure
• Administer 2 infusions in anterior putamen and 2 infusions in posterior putamen

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Eladocagene Exuparvovec has no noted severe interactions with any other drugs
• Eladocagene Exuparvovec has no noted serious interactions with any other drugs
• Eladocagene Exuparvovec has no noted moderate interactions with any other drugs
• Eladocagene Exuparvovec has no noted minor interactions with any other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• Pediatric patients who have not achieved skull maturity, assessed by neuroimaging

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Eladocagene Exuparvovec?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Eladocagene Exuparvovec?”

Cautions

• Procedural complications
  • Complications (e.g., respiratory and cardiac arrest) reported after the procedure
  • Events occurred within 24 hours of neurosurgical procedure and during post-surgical care
• Other risks include
• Cerebrospinal fluid (CSF) leak
• Intracranial bleeding
• Neuroinflammation
• Acute infarction
• Infection
  • Monitor for procedure-related adverse events during administration; use continuous cardiorespiratory monitoring during hospitalization
• Dyskinesia
  • Can cause dyskinesia (e.g., involuntary movements of face, arm, leg, or entire body)
  • Events reported within 3 months of administration; 2 cases required hospitalization
  • Monitor for signs and symptoms of dyskinesia (e.g., fidgeting, writhing, wriggling, head bobbing, body swaying) after treatment
  • Use of dopamine antagonists may be considered to control dyskinesia symptoms

Pregnancy and Lactation

• No clinical data in pregnant women or from animal reproductive and developmental toxicity studies to assess risk during pregnancy
• Verify negative pregnancy test before therapy in females of reproductive potential
• No recommendation for the duration of contraception following therapy due to insufficient exposure data
• Infertility
  • No data regarding the effects on fertility
• Lactation
  • No data are available regarding the presence of Eladocagene Exuparvovec in human milk, its effects on breastfed infants, or milk production