Currentculinary health fundPolicy N/A

Amyloidosis - Onpattro (patisiran) Utilization Management Medical Policy

Defines prior authorization, dosing, and coverage criteria for Onpattro (patisiran IV) for treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults; applies to medical benefit requests reviewed by the payer.

Policy Summary

Payerculinary health fund

PolicyAmyloidosis - Onpattro (patisiran) Utilization Management Medical Policy

Policy CodePolicy N/A

Change TypeAdded exclusion, clarified terminology, expanded concurrent-therapy list

Effective Date

Next Review DateDec 10, 2025

Key ActionObtain prior authorization and document genetic testing confirming a transthyretin pathogenic variant, symptomatic polyneuropathy, age ≥18, absence of prior liver transplant, and specialist involvement.

SourceLink

POLICY UPDATE CHANGES

The patient does not have a history of liver transplant was added as an approval requirement.

Language for diagnosis confirmation changed from 'mutation' to 'pathogenic variant' for genetic testing confirmation.

Conditions Not Recommended for Approval updated to list concurrent use with other indicated agents (Amvuttra, Attruby, Tegsedi, Wainua, or a tafamidis product).

≥18minimum age for approval

0.3 mg/kgrecommended IV dosing

q3wdosing interval

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Recommended Authorization Criteria

Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

FDA-Approved Indication Criteria: Patient is >= 18 years of age; AND has a transthyretin pathogenic variant confirmed by genetic testing; AND has symptomatic polyneuropathy; AND does not have a history of liver transplantation; AND medication is prescribed by or in consultation with a neurologist, geneticist, or physician who specializes in amyloidosis.NA

Examples of symptomatic polyneuropathy assessments include history/clinical exam, electromyography (EMG), or nerve conduction velocity (NCV) testing.

Dosing Requirements: Dose up to 0.3 mg/kg IV (maximum 30 mg) administered no more frequently than once every 3 weeks.0.3 mg/kg; max 30 mg; interval >= 3 weeks

Approve for up to 1 year when all clinical criteria and dosing requirements are met.

Coverage of Onpattro (patisiran) in combination with other agents indicated for hereditary transthyretin-mediated amyloidosis is not recommended. Specifically, concurrent use with Amvuttra (vutrisiran), Attruby (acoramidis), Tegsedi (inotersen), Wainua (eplontersen), or any tafamidis product should not be approved. The policy states that the requested medication should not be administered in combination with other medications indicated for polyneuropathy of hATTR or transthyretin-mediated amyloidosis–cardiomyopathy because combination therapy is generally not recommended due to a lack of controlled clinical trial data supporting additive efficacy.

Coverage is not recommended for circumstances not explicitly listed in the Recommended Authorization Criteria. Requests that do not meet the specific approval criteria set forth by the policy — including diagnostic, age, transplant history, specialist prescriber, and dosing requirements — should not be approved. The criteria will be updated as new published data become available.

Drug Dosing and Codes

Covered NDC/Drug DosingNDCCovered

NDC	Onpattro (patisiran) intravenous infusion dosing up to 0.3 mg/kg (max 30 mg) every 3 weeks

inv-05: Dose limit — 0.3 mg/kg, maximum 30 mg per dose

Dose limit0.3 mg/kg per infusion (maximum 30 mg per dose)

Dosing routeIntravenous (IV) administration

Approval noteDose limit used to determine medical necessity and to calculate maximum per-infusion quantity

inv-06: Minimum dosing interval — Not more frequently than once every 3 weeks

Minimum dosing intervalNot more frequently than once every 3 weeks

Implication for schedulingUse 3-week minimum spacing when authorizing repeat infusions

Authorization, Documentation, and Exclusions

Prior Authorization

Prior Authorization Required

Prior authorization is required for Onpattro. Approvals will be for up to 1 year when the coverage criteria are met. Because of the specialized skills required for evaluation, diagnosis, and monitoring, Onpattro must be prescribed by or in consultation with a neurologist, geneticist, or a physician who specializes in the treatment of amyloidosis.

Prior authorization required
Approval duration: up to 1 year when criteria met
Prescriber requirement: neurologist, geneticist, or amyloidosis specialist

Step Therapy

Step Therapy / Specialist Requirement

No step therapy (no requirement to try alternative agents first) is mandated in this policy; however, the medication must be prescribed by or in consultation with a specialist as described in the criteria.

No explicit step-therapy sequence required
Specialist involvement required (neurologist, geneticist, or amyloidosis specialist)

Clinical Background

Hereditary transthyretin-mediated amyloidosis (hATTR) is a progressive, inherited disorder caused by pathogenic variants in the transthyretin (TTR) gene that leads to multisystem organ dysfunction. Neurologic manifestations commonly include sensorimotor and autonomic polyneuropathy, which may be documented by clinical history and exam or electrodiagnostic testing (EMG/NCV). Onpattro (patisiran) is an RNA interference therapeutic indicated for adult patients with hATTR polyneuropathy and has demonstrated reductions in TTR and improvements in neuropathy and quality-of-life measures.

Key Definitions

inv-14: Symptomatic polyneuropathy — clinical manifestations definition

DefinitionClinically evident neuropathy manifested by reduced motor strength or coordination, or impaired sensation (e.g., pain, temperature, vibration, touch)

Assessment methodsDocumented by history and clinical exam or electrodiagnostic testing such as electromyography (EMG) or nerve conduction velocity (NCV)

Common manifestationsIncludes sensorimotor and autonomic polyneuropathy, small-fiber neuropathy, and related symptoms

inv-15: Transthyretin pathogenic variant — genetic test-confirmed definition (language updated from 'mutation')

DefinitionA transthyretin (TTR) gene pathogenic variant confirmed by genetic testing

Terminology notePolicy language uses 'pathogenic variant' instead of 'mutation' for diagnostic confirmation