CurrentCentenePolicy CP PHAR.395

Patisiran (Onpattro)

Defines medical necessity, prior authorization criteria, dosing limits, and continuity/renewal requirements for Onpattro (patisiran) for members covered under Centene-affiliated health plans (Commercial, Medicaid, HIM - Medical Benefit).

Policy Summary

PayerCentene

PolicyPatisiran (Onpattro)

Policy CodePolicy CP PHAR.395

Change TypeAdded and removed concurrent-use/prior treatment criteriaclarified commercial approval duration

Effective DateSep 11, 2018

Next Review Date

Key ActionSubmit supporting documentation (office notes, lab results, biopsy reports, and genetic sequencing) to demonstrate the member meets all approval criteria, including genetic confirmation or justification if biopsy is absent or negative.

SourceLink

POLICY UPDATE CHANGES

Added Wainua to the list of drugs that should not be prescribed concurrently (2Q 2024).

Removed requirement that member has not received treatment with Amvuttra, Tegsedi, or Wainua (2Q 2025).

Clarified approval duration for commercial line of business injectable drug: 6 months or to the member's renewal date, whichever is longer.

Adults onlyAge group eligible

Weight-basedDosing regimens

6 / 12Approval durations

J0222

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Medical Necessity Criteria for Onpattro (patisiran)

Initial Approval — Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Onpattro is medically necessary when ALL of the following are met:

ALL of the following

Diagnosis of hereditary TTR-mediated amyloidosis with polyneuropathy
Documentation confirms presence of a transthyretin (TTR) mutation
DNA sequencing is usually required for genetic confirmation; techniques detect ~99% of disease-causing mutations
Prescribed by or in consultation with a neurologist
Biopsy is positive for amyloid deposits OR medical justification provided to initiate treatment despite a negative biopsy or absence of biopsy
Demonstration of amyloid via Congo red staining recommended; negative biopsy does not exclude disease
Age > 18 years
Member has not had a liver transplant
Prior liver transplant excludes approval
Dose limits (must not exceed)
- Weight < 100 kg: 0.3 mg/kg IV once every 3 weeksWeight < 100 kg
- Weight ≥ 100 kg: 30 mg IV once every 3 weeksWeight ≥ 100 kg
Onpattro is not prescribed concurrently with Amvuttra, Tegsedi, or Wainua
Concurrent-prescription exclusion existed in earlier versions; later removal noted in 2Q 2025 changes

Provider must submit supporting documentation (office notes, lab results, biopsy reports) demonstrating member meets all approval criteria including genetic confirmation and biopsy results or justification for lack of biopsy.

HCPCS J0222 is the billing code for patisiran (0.1 mg)

Initial Approval — Other diagnoses/indications

Requests not meeting the specific initial criteria follow other policies (must meet either 1 or 2):

ANY of the following

For drugs on the formulary/PDL with a recent label change (within 6 months), refer to commercial formulary/non-formulary or PDL policies: CP.CPA.190 (commercial), HIM.PA.33 (HIM), CP PMN.255 (Medicaid)
If requested use is not listed and no recent label change applies, refer to off-label use policies: CP.CPA.09 (commercial), HIM.PA.154 (HIM), CP PMN.53 (Medicaid)
If drug is not on formulary/PDL, refer to non-formulary policies: CP.CPA.190 (commercial), HIM.PA.103 (HIM), CP PMN.16 (Medicaid)

Continuation (Renewal) Criteria — Hereditary Transthyretin-Mediated Amyloidosis

For continued therapy, member must meet ALL of the following:

ALL of the following

Continuity/previous approval
- Member is currently receiving medication via Centene benefit
- Member has previously met initial approval criteria
Member is responding positively to therapy
Examples: improved measures of polyneuropathy (motor strength, sensation, reflexes), quality of life, motor function, walking ability (e.g., timed 10-m walk test), and nutritional status (e.g., modified mass index)
Onpattro is not prescribed concurrently with Amvuttra, Tegsedi, or Wainua
Concurrent-prescription exclusion noted in policy history; see changes for 2025 removal

Continued Therapy — Other diagnoses/indications

Same process as initial Other diagnoses/indications: refer to formulary/off-label/non-formulary policies as appropriate

ANY of the following

Refer to CP.CPA.190, HIM.PA.33, CP PMN.255 for formulary-related exceptions or recent label changes
Refer to CP.CPA.09, HIM.PA.154, CP.PMN.53 for off-label requests not listed
Refer to CP.CPA.190, HIM.PA.103, CP PMN.16 for non-formulary requests

Diagnoses/Indications Not Authorized

Coverage is NOT authorized for:

ANY of the following

Non-FDA approved indications not addressed in this policy unless sufficient documentation per off-label use policies demonstrates efficacy and safety

Relevant Billing Codes

HCPCS - J-code for patisiranHCPCS

J0222

Injection, patisiran, 0.1 mg

Prior Authorization, Documentation, and Billing Rules

Prior Authorization

Submit documentation to support criteria

Provider must submit supporting documentation (such as office chart notes, lab results, or biopsy reports) that demonstrates the member meets all approval criteria, including genetic confirmation and biopsy results or a medical justification for lack of biopsy. Include HCPCS code J0222 on submissions for patisiran dosing/administration claims.

Relevant documentation: office notes, laboratory results, biopsy reports
Include HCPCS code J0222 for patisiran (Injection, patisiran, 0.1 mg) when applicable

Documentation Required

Genetic confirmation

Provide documentation confirming a TTR mutation via DNA sequencing; appendix states DNA sequencing is usually required for genetic confirmation and current techniques detect ~99% of disease-causing mutations.

Key Thresholds and Approval Durations

Approval thresholds and durations

Age threshold> 18 years

Dose threshold - weight basedWeight < 100 kg: 0.3 mg/kg IV every 3 weeks; Weight ≥ 100 kg: 30 mg IV every 3 weeks

Initial approval duration (Medicaid/HIM)6 months

Initial approval duration (Commercial)6 months or to the member's renewal date, whichever is longer

Continued approval duration (Medicaid/HIM)12 months

Continued approval duration (Commercial)6 months or to the member's renewal date, whichever is longer

Clinical Background

Patisiran (Onpattro) is an RNA interference therapeutic directed at transthyretin (TTR) used to treat the polyneuropathy of hereditary TTR‑mediated amyloidosis in adults. The agent is a TTR‑directed small interfering ribonucleic acid designed to reduce pathogenic TTR protein produced by mutant TTR genes, and its FDA‑approved indication is treatment of the polyneuropathy of hereditary TTR‑mediated amyloidosis in adults.

Genetic testing to document a pathogenic TTR mutation is central to diagnosis; the policy notes that DNA sequencing is usually required for genetic confirmation and that current sequencing techniques detect approximately 99% of disease‑causing mutations. Tissue biopsy demonstrating amyloid deposits by Congo red staining is also recommended to confirm amyloidosis—Congo red‑positive deposits show characteristic birefringence under polarized light—but the policy explicitly states that a negative biopsy does not exclude the disease, and clinical justification may support treatment despite negative or absent biopsy results.

Defined Terms

Definitions

TTRTransthyretin, the protein associated with hereditary transthyretin-mediated amyloidosis.

OnpattroBrand name for patisiran, a TTR-directed small interfering ribonucleic acid therapeutic.

Policy Changes

2024-01-09added

Wainua was added to the policy as a drug that should not be prescribed previously or concurrently with Onpattro (concurrent-use exclusion).

2025-01-17removedLatest

Requirement that members have not received treatment with Amvuttra, Tegsedi, or Wainua was removed to allow these prior treatments as alternatives following competitor analysis and market changes.

2022-11-22revised

Clarified approval duration for commercial line of business injectable drugs to be 6 months or to the member's renewal date, whichever is longer (operational clarification).

Patisiran (Onpattro)

Medical Necessity Criteria for Onpattro (patisiran)

Relevant Billing Codes

Prior Authorization, Documentation, and Billing Rules

Key Thresholds and Approval Durations

Clinical Background

Defined Terms

Other Policies to Reference

Policy Changes