Last Review Date: 12/03/2024

Date of Origin: 12/03/2024

Dates Reviewed: 12/2024

1. Length of Authorization

Coverage will be provided for one dose and may not be renewed.

2. Dosing Limits

A. Max Units (per dose and over time) [HCPCS Unit]:

• 1.8x10¹¹ vg (0.32 mL) one time only

3. Initial Approval Criteria ^1-15

Coverage is provided in the following conditions:

Aromatic L-amino acid decarboxylase (AADC) deficiency

• Patient is at least 16 months of age through 10 years of age; AND
• Patient has a diagnosis of severe Aromatic L-amino acid decarboxylase (AADC) deficiency as established by the following:
  • Patient has a biallelic pathogenic variants in DDC gene identified by molecular genetic testing; OR
  • Patient cerebrospinal fluid (CSF) or plasma neurotransmitter profile is consistent with AADC deficiency; AND
    • Patient has significantly reduced AADC enzyme activity in plasma.; AND
• Patient is experiencing persistent neurological defects (e.g., autonomic dysfunction, hypotonia, dystonia and other movement disorders, etc.) secondary to AADC deficiency despite standard medical therapy (e.g., dopamine agonists, monoamine oxidase inhibitor, pyridoxine, or other forms of vitamin B6) Note: patients should be on stable dosages for at least 3 months prior to treatment with eladocagene; AND
• Patient is unable to ambulate independently; AND
• Patient has achieved skull maturity as assessed by neuroimaging; AND
• Patient does not have pyridoxine 5'-phosphate oxidase or tetrahydrobiopterin (BH4) deficiency; AND
• Patient has not received prior gene therapy; AND
• Patient must not have a baseline anti-AAV2 antibody titer above the established threshold for a positive result; AND
• Patient does not have any contraindications that would preclude the surgical intra-putaminal administration

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ¹

Coverage cannot be renewed.

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS code:

• J3590 – Unclassified biologics

NDC:

• Kebilidi 5.6 × 10¹¹ vector genomes (vg) per mL – 2 mL single dose vial: 52856-0601-xx

7. References

1. Kebilidi [package insert]. Warren, NJ; PTC Therap., Inc., November 2024. Accessed November 2024.
2. ClinicalTrials.gov. NCT01395641. A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC. https://clinicaltrials.gov/study/NCT01395641 .
3. ClinicalTrials.gov. NCT02926066. A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion (NTUH-AADC-011). https://clinicaltrials.gov/study/NCT02926066 .
4. Blau N, Pearson TS, Kurian MA, et al. Aromatic L-Amino Acid Decarboxylase Deficiency. GeneReviews. https://www.ncbi.nlm.nih.gov/books/NBK595821/ (Accessed on August 5, 2024).
5. Wassenberg T, Molero-Luis M, Jeltsch K, et al. Consensus guideline for the diagnosis and treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. Orphanet J Rare Dis. 2017. https://doi.org/10.1186/s13023-016-0522-z.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCA/LCD): N/A