Last Review Date: 07/02/2024

Date of Origin: 09/05/2018

Dates Reviewed: 09/2018, 10/2019, 10/2020, 10/2021, 10/2022, 10/2023, 07/2024

1. Length of Authorization

Coverage will be provided for 12 months and may be renewed annually thereafter.

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• Onpattro 10 mg/5ml single-dose vial injection: 3 vials every 3 weeks

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

• 300 billable units every 3 weeks

3. Initial Approval Criteria ¹

          Coverage is provided in the following conditions:

• Patient is at least 18 years of age; AND

Universal Criteria ¹

• Patient is receiving supplementation with vitamin A at the recommended daily allowance; AND
• Must not be used in combination with other transthyretin (TTR) reducing or stabilizing agents (e.g., inotersen, tafamidis, vutrisiran, etc.); AND

Polyneuropathy due to Hereditary Transthyretin-Mediated (hATTR) Amyloidosis † Ф ^1-3,5,6

• Patient has a definitive diagnosis of hATTR amyloidosis as documented by one of the following:
  • Amyloid deposition on tissue biopsy; OR
  • Identification of a pathogenic TTR variant using molecular genetic testing; AND
• Used for the treatment of polyneuropathy as demonstrated by at least TWO of the following criteria:

• Subjective patient symptoms are suggestive of neuropathy
• Abnormal nerve conduction studies are consistent with polyneuropathy
• Abnormal neurological examination is suggestive of neuropathy; AND

• Patient’s peripheral neuropathy is attributed to hATTR and other causes of neuropathy have been excluded; AND
• Baseline in strength/weakness has been documented using an objective clinical measuring tool (e.g., Medical Research Council (MRC) muscle strength, etc.); AND
• Patient has not been the recipient of an orthotopic liver transplant (OLT)

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

4. Renewal Criteria ^1-3,5,6

Coverage can be renewed based upon the following criteria:

• Patient continues to meet the universal and other indication-specific relevant criteria identified in section III; AND
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: severe infusion-related reactions, ocular symptoms related to vitamin A deficiency (e.g., night blindness), etc.; AND
• Disease response compared to pre-treatment baseline as evidenced by stabilization or improvement in one or more of the following:

• Signs and symptoms of neuropathy
• MRC muscle strength

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS Code:

• J0222 - Injection, patisiran, 0.1 mg; 1 billable unit = 0.1 mg

NDC:

• Onpattro 10 mg/5 mL single-dose vial: 71336-1000-xx

7. References

1. Onpattro [package insert]. Cambridge, MA; Alnylam Pharmaceuticals, Inc., January 2023. Accessed May 2024.
2. Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153
3. Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017;17(1):181
4. Ando Y, Coelho T, Berk JL, et al. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis. 2013;8:31.
5. Sekijima Y, Nakamura K. Hereditary Transthyretin Amyloidosis. In: Adam MP, Feldman J, Mirzaa G, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024. Initial Posting: November 5, 2001; Last Update: May 30, 2024. Accessed June 12, 2024. https://www.ncbi.nlm.nih.gov/books/NBK1194/.
6. Luigetti M, Romano A, DiPaolantonio A, et al. Diagnosis and Treatment of Hereditary Transthyretin Amyloidosis (hATTR) Polyneuropathy: Current Perspectives on Improving Patient Care. Ther Clin Risk Manag. 2020; 16: 109–123.Published online 2020 Feb 21. doi: 10.2147/TCRM.S219979.

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