Medical Drug Criteria (MDC)

Policy Num:       P1.002.017
Policy Name:    Kebilidi (Eladocagene exuparvovec-tneq)
Policy ID:          [P1.002.017] [Ac/L/ M+/ P+ ]

Last Review:       September 22, 2025
Next Review:      September 20, 2025
 

Related MDC:

KEBILIDI (Eladocagene exuparvovec-tneq)

Summary

KEBILIDI is an adeno-associated virus (AAV) vector-based gene therapy 12 indicated for the treatment of adult and pediatric patients with aromatic 13 L-amino acid decarboxylase (AADC) deficiency.

objective

Define medical necessity and coverage criteria for Kebilidi under Triple-S health plans and aligning with FDA labeling.

Policy Statements

Coverage for Kebilidi is medically necessary when all of the following are met:

1. Confirmed diagnosis of AADC deficiency due to biallelic DDC mutations (genetic report required).

2. Documented skull maturity by neuroimaging (contraindicated if absent).

3. Administration at a specialized center with expertise in stereotactic neurosurgery and use of an FDA-authorized cannula for intraparenchymal infusion. 

4. For women of reproductive potential: a documented negative pregnancy test prior to infusion.

Policy Guidelines

Required Medical Documentation
 
Genetic report confirming DDC pathogenic variants; plasma AADC activity result.
 
Neurology evaluation documenting phenotype and prior medical therapy trials/response.
 
Neuroimaging confirming skull maturity and surgical planning suitability.
 
Anti-AAV2 antibody result and perioperative plan (ICU/step-down, MRI access).

DOSAGE/ADMINISTRATION

Route/target: Stereotactic intracerebral intraputaminal infusion.
 
Total dose: 1.8×10¹¹ vg (0.32 mL) delivered as 4 infusions (0.08 mL; 0.45×10¹¹ vg per site) at a rate of 0.003 mL/min (~27 minutes per site).

Formulation: Suspension 5.6×10¹¹ vg/mL; single-use vial.

REQUIRED MEDICAL INFORMATION

1. Genetic confirmation of biallelic DDC mutations and clinical documentation. 
 
2. Neuroimaging confirming skull maturity and surgical planning.
  
3. Surgical plan and confirmation of FDA-cleared delivery device. 
 
4. Negative pregnancy test for women of childbearing potential.
 
5. Pre-anesthetic clearance and perioperative monitoring plan.

EXCLUSION CRITERIA

No confirmed biallelic DDC mutation.
 
Lack of skull maturity (contraindicated).
 
Use for any off-label indication.

OTHER CRITERIA

None

Practice Guidelines and Position Statements

iNTD Consensus (2017): Diagnostic triad (biochemical CSF pattern, DDC genotyping, ↓ plasma AADC), stepwise symptomatic management (dopamine agonists, MAO-Is, pyridoxine), and multidisciplinary care. Gene therapy is emerging for severe phenotypes.
 

References

1. FDA | CBER Product Page – KEBILIDI. Indication and product details. 
 
2. FDA Prescribing Information – KEBILIDI (PI, Nov 2024). Mechanism, dose, administration, safety. 
 
3. UnitedHealthcare Medical Benefit Drug Policy (Aug 1, 2025). Coverage rationale, criteria, codes.
 
4. Medscape Drug Reference – Kebilidi. Dosage summary and administration overview.
 
5. Clinical Therapeutics (2025). Device note: SmartFlow Neuro Cannula indicated for Kebilidi administration. 
 
6. FDA Press / Company Releases (Nov 2024). Approval announcements and pathway.

Codes

Applicable Modifiers

Policy History