CurrentmhnPolicy CP.PHAR.395

Patisiran (Onpattro)

Defines medical necessity criteria, dosing limits, concurrent therapy exclusions, approval durations, and documentation requirements for coverage of patisiran (Onpattro) for hereditary transthyretin-mediated amyloidosis with polyneuropathy across Commercial, Medicaid, and HIM/ICHRA lines of business.

Policy Summary

Payermhn

PolicyPatisiran (Onpattro)

Policy CodePolicy CP.PHAR.395

Change TypeMultiple annual updates (added/removed/revised criteria)

Effective DateSep 11, 2018

Next Review Date

Key ActionProvider must submit documentation supporting that member meets all approval criteria.

SourceLink

POLICY UPDATE CHANGES

Added Wainua to list of drugs that should not have been previously received or prescribed concurrently.

Removed criteria requiring no prior treatment with Amvuttra, Tegsedi, or Wainua.

Revised initial approval duration for Medicaid/HIM from 6 to 12 months and added ICHRA line of business.

Added concurrent use exclusion with Amvuttra and Tegsedi (historical) and no prior liver transplant criterion in prior revisions.

1FDA-approved Indications

18+Minimum Age (years)

2Approval Durations Listed

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Coverage Summary

Patisiran (Onpattro) is covered with criteria for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy across Commercial, Medicaid, and HIM/ICHRA lines of business. Coverage requires meeting the policy's medical necessity criteria, including documentation of diagnosis, neurologist involvement, weight-based dosing limits, exclusions for concurrent therapy, and other requirements. The policy specifies a minimum age of 18 years for treatment. Dosing limits are weight-based (0.3 mg/kg every 3 weeks if weight < 100 kg; 30 mg every 3 weeks if weight ≥ 100 kg). Concurrent prescription with Amvuttra or Wainua is not allowed, and members must not have had a prior liver transplant. Initial and continuation approval durations vary by line of business: for Medicaid/HIM/ICHRA the initial approval duration is 12 months, while for Commercial it is 6 months or to the member's renewal date, whichever is longer (approval durations and some specific requirements may differ between initial and renewal criteria).

Initial Therapy Criteria - Hereditary Transthyretin-Mediated Amyloidosis

Initial Approval Criteria - Hereditary Transthyretin-Mediated Amyloidosis

Covered when ALL of the following are met:

ALL of the following

Diagnosis of hereditary TTR-mediated amyloidosis with polyneuropathy
Genetic confirmation: Documentation confirms presence of a TTR mutation
DNA sequencing is usually required for genetic confirmation per policy appendix
One of the following
- Biopsy positive: Biopsy is positive for amyloid deposits
  Congo red staining and characteristic green birefringence under polarized light described in policy appendix
- Justification: Medical justification is provided as to why treatment should be initiated despite a negative biopsy or no biopsy
  Negative biopsy should not exclude disease per policy appendix
Prescriber: Prescribed by or in consultation with a neurologist
Age ≥ 18 years>= 18 years
Transplant: Member has not had a prior liver transplant
Concurrent therapy exclusion: Onpattro is not prescribed concurrently with Amvuttra or Wainua
Requests will be denied if prescribed concurrently per provider actions
Dose limits
- Weight < 100 kg: 0.3 mg/kg once every 3 weeks0.3 mg/kg q3 weeks if weight < 100 kg
- Weight ≥ 100 kg: 30 mg once every 3 weeks30 mg q3 weeks if weight >= 100 kg

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Initial Therapy — Other Diagnoses / Indications

Initial Approval Criteria - Other diagnoses/indications

If requested use is not covered under the above and one of the following applies:

ANY of the following

Recent label change pathway: If drug has recently (within last 6 months) undergone a label change not yet reflected in this policy, follow formulary/non-formulary pathways
Refer to CP.CPA.190, HIM.PA.33, CP.PMN.255 for formulary; CP.CPA.190, HIM.PA.103, CP.PMN.16 for non-formulary as applicable
Off-label pathway: If requested use is not listed in section III and criterion above does not apply, refer to off-label use policy
Refer to CP.CPA.09, HIM.PA.154, CP.PMN.53 as applicable

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Continuation (Renewal) Criteria

Continuation (Renewal) Criteria - Hereditary TTR-mediated amyloidosis

Covered when ALL of the following are met:

ALL of the following

Member must meet one of
- Current Centene benefit or prior initial approval: Currently receiving medication via Centene benefit or previously met initial approval criteria
- Continuity-of-care enrollment: Member is currently receiving medication and is enrolled in a state/product with continuity of care regulations
  Refer to CC.PHARM.03A and CC.PHARM.03B state addendums
Clinical response:

Diagnoses / Indications Not Authorized

Diagnoses/Indications Not Authorized

Coverage is NOT authorized for:

Non-FDA approved indications not addressed in this policy

Not authorized unless sufficient documentation of efficacy and safety per off-label use policies or evidence of coverage documents

Applicable Codes

HCPCSHCPCS

J0222

Injection, patisiran, 0.1 mg

Provider Actions

Prior Authorization

Prior authorization required

Prior authorization is required for Onpattro (patisiran). Submit a prior authorization request referencing HCPCS code J0222. Initial and continuation approval durations vary by line of business (Medicaid/HIM/ICHRA: 12 months; Commercial: 6 months or to the member's renewal date, whichever is longer).

Code: J0222 (Injection, patisiran, 0.1 mg)
Approval durations: Medicaid/HIM/ICHRA 12 months; Commercial 6 months or to member renewal date, whichever is longer

Documentation Required

Submit supporting clinical documentation

Submit supporting clinical documentation with the prior authorization request. Acceptable documentation includes office chart notes, laboratory results, and other clinical information that demonstrates the member meets all approval criteria.

Office notes

Clinical Evidence

Key evidence includes the pivotal randomized controlled trial: Adams et al., N Engl J Med. 2018 demonstrating the efficacy of patisiran. The policy also references the product Prescribing Information: Onpattro Prescribing Information, Alnylam Pharmaceuticals (document listed with access date January 14, 2026). Full citations are provided in the policy evidence/references section.

Background

Background: Patisiran is a transthyretin (TTR)-directed small interfering RNA indicated for treatment of the polyneuropathy of hereditary TTR-mediated amyloidosis in adults. Diagnostic confirmation of TTR amyloidosis relies on demonstration of amyloid deposits via tissue biopsy using Congo red staining and genetic confirmation by DNA sequencing of the TTR gene; negative biopsy results do not exclude the disease and medical justification may be provided if biopsy is negative or not performed. Definitions: TTR = transthyretin.

Revision History

08.01.22 / P&T 11.22added

4Q 2022 annual review: added criterion for no prior treatment with Amvuttra or Tegsedi; updated concurrent use exclusion with Amvuttra for initial and continued approval criteria; clarified commercial approval duration and applied template changes.

01.09.24 / P&T 05.24added

2Q 2024 annual review: added Wainua to the list of drugs that should not have been previously received or prescribed concurrently; references reviewed and updated.

01.17.25 / P&T 05.25removed