CurrentCommunity-CarePolicy N/A

Onpattro (patisiran) — Medical Benefit Medication Utilization Policy

Defines medical benefit coverage, initial and continuation criteria, quantity limits, and coding for patisiran (Onpattro) for treatment of polyneuropathy due to hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Policy Summary

PayerCommunity-Care

PolicyOnpattro (patisiran) — Medical Benefit Medication Utilization Policy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateSep 1, 2024

Key ActionObtain prior authorization with documentation confirming specialist prescriber, confirmed hATTR diagnosis (biopsy or specified non-invasive testing and genetic sequencing), baseline neuropathy score, and patient weight.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥18minimum patient age for coverage

1 yearauthorization period

J0222HCPCS code

weight-basedquantity limits

Coverage Criteria for Patisiran (Onpattro)

Initial Therapy

Covered when ALL of the following are met for initial approval:

Initial Approval - hATTR polyneuropathy: 1) Prescribed by a neurologist, cardiologist, hematologist, or other specialist experienced in the diagnosis and treatment of hereditary transthyretin-mediated (hATTR) amyloidosis; 2) Diagnosis of polyneuropathy of hATTR amyloidosis; 3) Age ≥ 18 years; 4) Amyloidosis confirmed by either: a positive tissue biopsy OR non-invasive testing demonstrating ALL of: bone scintigraphy with myocardial uptake > 1.5 heart-to-contralateral ratio or grade >2 visual evidence; serum kappa/lambda free light chain ratio 0.26–1.65; absence of monoclonal protein on serum immunofixation; absence of monoclonal protein on urine immunofixation; 5) hATTR confirmed by genetic sequencing; 6) Polyneuropathy confirmed by either: baseline Polyneuropathy Disability (PND) score ≥ 1 OR baseline Familial Amyloidosis Polyneuropathy (FAP) Stage ≥ 1 with at least one of: autonomic dysfunction; characteristic symmetrical length-dependent peripheral neuropathy beginning in lower limbs and progressing proximally; bilateral carpal tunnel syndrome; or cardiac symptoms; 7) Will not be used in combination with other TTR silencing or stabilizing agents (including but not limited to tafamidis, eplontersen, vutrisiran).See node text

All listed conditions must be satisfied for initial coverage.

Continuation Therapy

Covered for continuation when ALL of the following are met:

Policy Summary

PayerCommunity-Care

PolicyOnpattro (patisiran) — Medical Benefit Medication Utilization Policy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateSep 1, 2024

SourceLink

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Provider Actions, Documentation, and Restrictions

Prior Authorization

Prior authorization required — meet all initial approval criteria

Prior authorization is required and will only be approved when the initial approval criteria are met, including that the drug is prescribed by a neurologist, cardiologist, hematologist, or other specialist experienced in hATTR; diagnosis of polyneuropathy of hATTR amyloidosis; age ≥ 18 years; amyloidosis confirmation (tissue biopsy or specified non‑invasive testing); hATTR confirmed by genetic sequencing; and polyneuropathy baseline (PND ≥1 or FAP Stage ≥1 with qualifying features). If approved, weight-based quantities (J0222) will be authorized.

Step Therapy

Combination therapy restriction — no concurrent TTR agents

Do not prescribe patisiran in combination with other transthyretin (TTR) silencing or stabilizing agents; combination use (including but not limited to tafamidis, eplontersen, vutrisiran, or inotersen) is not allowed for initial or continuation therapy.

Documentation Required

Required documentation for initial approval — specialist, diagnosis, confirmations, baseline scores, weight

Provide documentation with the prior authorization request that includes the specialist prescriber type, diagnosis of polyneuropathy of hATTR amyloidosis, patient age (≥18), confirmation of amyloidosis by positive tissue biopsy or the specified non‑invasive testing criteria, genetic sequencing confirming hATTR, baseline neuropathy score (PND ≥1 or FAP Stage ≥1 with qualifying features), and patient weight for quantity determination.

Denial Risk

Insufficient diagnostic confirmation may trigger denial or require more information

If hATTR is not confirmed by genetic sequencing or amyloidosis is not confirmed by a tissue biopsy or by the specified non‑invasive testing (bone scintigraphy uptake >1.5 heart‑to‑contralateral ratio or grade >2 visual evidence; serum kappa/lambda ratio 0.26–1.65; absence of monoclonal protein on serum and urine immunofixation), the request may be denied or additional documentation will be required.

Definitions and Diagnostic Confirmation

Amyloidosis confirmation — Biopsy or specified non‑invasive testing

Acceptable tissue confirmationPositive tissue biopsy confirming amyloidosis

Acceptable non‑invasive confirmation — imagingBone scintigraphy with myocardial uptake > 1.5 heart‑to‑contralateral ratio or grade > 2 visual evidence

Acceptable non‑invasive confirmation — laboratorySerum kappa/lambda free light chain ratio 0.26–1.65 AND absence of monoclonal protein on serum and urine immunofixation

Polyneuropathy confirmation — Baseline PND or FAP Stage and qualifying features

PND thresholdBaseline Polyneuropathy Disability (PND) score ≥ 1

FAP thresholdBaseline Familial Amyloidosis Polyneuropathy (FAP) Stage ≥ 1

Required accompanying features for FAP ≥1At least one of: autonomic dysfunction; symmetrical length‑dependent peripheral neuropathy beginning in lower limbs and progressing proximally; bilateral carpal tunnel syndrome; or cardiac symptoms

Background

Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive systemic disorder that can cause debilitating peripheral polyneuropathy and cardiac involvement. Patisiran (Onpattro) is an RNA interference therapeutic indicated for the treatment of polyneuropathy of hATTR amyloidosis and is intended for management by clinicians experienced in diagnosing and treating hATTR.

Prior to authorization, diagnostic confirmation is required (see definitions and coverage criteria). Age for coverage is ≥ 18 years, and baseline neuropathy must be documented (for example, a baseline Polyneuropathy Disability score of ≥ 1 or Familial Amyloidosis Polyneuropathy Stage of ≥ 1 with qualifying features). If approved, dosing quantities are weight-based and supplied as 0.1 mg vials (HCPCS J0222); authorization periods are for 1 year.

OpenPayer

Onpattro (patisiran) — Medical Benefit Medication Utilization Policy

Coverage Criteria for Patisiran (Onpattro)

Coding and Clinical Baseline

Provider Actions, Documentation, and Restrictions

Definitions and Diagnostic Confirmation

Background