Eladocagene exuparvovec-tneq (Kebilidi) intraputaminal infusion coverage criteria
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Defines medical necessity, coverage limitations, and documentation requirements for eladocagene exuparvovec-tneq (Kebilidi) intraputaminal infusion for treatment of AADC deficiency for Blue Cross NC members.
Added revenue codes 0891 and 0892 as applicable to the policy HCPCS code(s).
Coverage Criteria
(untitled)
Covered when ALL of the following are met:
ALL of the following
- The patient is at least 16 months of age through 10 years of age;
- The patient has a diagnosis of severe aromatic L-amino acid decarboxylase (AADC) deficiency (medical record documentation required);
Diagnosis confirmation
- Molecular genetic testing demonstrating biallelic mutations in the human DOPA decarboxylase (DDC) gene (medical record documentation required);
- Decreased plasma AADC enzyme activity (medical record documentation required);
- The patient is experiencing persistent neurological defects (e.g., autonomic dysfunction, hypotonia, movement disorders [oculogyric crises, dystonia, hypokinesia], developmental delay) secondary to AADC deficiency despite standard medical therapy (e.g., dopamine agonists, monoamine oxidase inhibitors, pyridoxine, or other forms of vitamin B6) on stable dosages for at least 3 months prior to treatment with the requested agent (medical record documentation required);
- The patient is unable to ambulate independently (with or without assistive device) (medical record documentation required);
- The patient has achieved skull maturity as assessed by neuroimaging (medical record documentation required);
- The patient does NOT have any other enzyme deficiencies, including pyridoxine 5'-phosphate oxidase or tetrahydrobiopterin (BH4) deficiency (medical record documentation required);
- The patient does NOT have a baseline anti-adeno-associated virus, serotype 2 (anti-AAV2) antibody titer higher than 1:1200 or greater than 1 optical density value by enzyme-linked immunosorbent assay (medical record documentation required);
- The patient does NOT have evidence of a clinically active infection (medical record documentation required);
- The patient has NOT received any previous gene therapy, including the requested agent (medical record documentation required);
(untitled)
AND the following provider/dosing conditions must be met:
ALL of the following
- The prescriber is a specialist in the area of the patient's diagnosis (e.g., pediatric neurologist) or has consulted with a specialist in the area of the patient's diagnosis (medical record documentation required);
- The requested dose is within FDA labeled dosing for the requested indication, and the requested quantity does NOT exceed the maximum units allowed for the duration of approval (medical record documentation required).
- Duration of Approval: 90 days (one-time treatment per lifetime).
- FDA label dosing: Total recommended dose of 1.8 x 10^11 vector genomes (vg), administered as four intraputaminal infusions in a single stereotactic neurosurgical procedure (medical record documentation required).
Exclude other enzyme deficiencies: The patient must NOT have any other enzyme deficiencies (e.g., pyridoxine 5'-phosphate oxidase or tetrahydrobiopterin [BH4] deficiency). Medical record documentation confirming absence of these alternative diagnoses is required.
Not medically necessary when any of the following apply: prior receipt of any gene therapy (including the requested agent); presence of a clinically active infection; baseline anti-AAV2 antibody titer greater than 1:1200 or optical density above assay cutoff; or other contraindicating conditions per FDA labeling. Medical record documentation required for exclusions and prior treatments.
Coding and Billing
| 0891 | Special Processed Drugs - FDA Approved Cell Therapy (applicable revenue code) |
| 0892 | Special Processed Drugs - FDA Approved Gene Therapy (applicable revenue code) |
Provider Actions and Requirements
Prior Authorization and Dosing/Quantity Limits
Prior authorization is required for coverage of the requested therapy. The requested dose must be within FDA-labeled dosing for the indication and the requested quantity must not exceed the maximum units allowed for the duration of approval. Duration of approval is a one-time treatment per lifetime (90 days). Distribution from a specialty pharmacy provider may be required when coverage is available — contact Blue Cross NC to coordinate therapy.
Medical Record Documentation Required for Diagnosis Confirmation and Testing
Medical record documentation is required to confirm the diagnosis and to support that medical necessity criteria are met. Submit documentation demonstrating patient age, diagnosis of severe AADC deficiency, confirmation testing results, prior treatment history, functional status, skull maturity assessment, infection and antibody testing, prior gene therapy history, and prescriber specialty or consultation.
- Patient age (≥ 16 months through 10 years)
- Diagnosis of severe AADC deficiency
- Molecular genetic testing showing biallelic DDC mutations
- Decreased plasma AADC enzyme activity
- Documentation of persistent neurological defects despite standard therapy
- Documentation of inability to ambulate independently
- Neuroimaging demonstrating skull maturity
- Anti-AAV2 antibody titer results and infection screening
- Record confirming no prior gene therapy
- Prescriber specialty (e.g., pediatric neurologist) or specialist consultation
Missing Required Documentation for Diagnosis Confirmation Is Grounds for Denial
Failure to provide the required documentation is grounds for claim denial. Specifically, lack of genetic testing demonstrating biallelic DDC mutations or absence of documented decreased plasma AADC enzyme activity will result in non-coverage. Missing evidence of prior standard therapy failure, functional status, skull maturity, antibody/infection testing, or prescriber specialty consultation may also lead to denial.
- Missing molecular genetic testing documenting biallelic DDC mutations = denial risk
- Missing decreased plasma AADC enzyme activity result = denial risk
- Missing documentation of failure of standard medical therapy (≥ 3 months on stable doses) = denial risk
- Missing documentation of inability to ambulate independently, skull maturity, antibody titer, infection status, or specialist involvement = denial risk
Failure of Standard Therapy
The patient must have persistent neurological deficits attributable to AADC deficiency despite an adequate trial of standard medical therapy. Documentation must show the patient was on stable dosages of therapies such as dopamine agonists, monoamine oxidase inhibitors, pyridoxine, or other vitamin B6 forms for at least 3 months prior to the requested treatment.
- Examples of persistent neurological defects: autonomic dysfunction, hypotonia, movement disorders (oculogyric crises, dystonia, hypokinesia), developmental delay
- Standard medical therapy trial: ≥ 3 months on stable dosages (e.g., dopamine agonists, MAO inhibitors, pyridoxine)
Background
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, inherited disorder caused by biallelic mutations in the DDC gene leading to markedly reduced AADC enzyme activity. Affected children present with persistent neurological deficits such as hypotonia, autonomic dysfunction, movement disorders (for example oculogyric crises, dystonia, hypokinesia), and developmental delay. Diagnosis requires medical record documentation of severe AADC deficiency confirmed by molecular genetic testing demonstrating biallelic DDC mutations and decreased plasma AADC enzyme activity.
Definitions
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