Treatment of Hereditary Amyloidosis (Medicaid Expansion)

Policy ID: ME-I-201-005-02

Section: Injections

Effective Date: January 01, 2022

Revised Date: November 29, 2022

Revision Effective Date: January 01, 2023

Last Reviewed: November 29, 2022

Applies To: Medicaid Expansion Only

Description

Patisiran (Onpattro^TM) is a RNA interference (RNAi) therapeutic targeting transthyretin for the treatment of hereditary transthyretin amyloidosis (TTR amyloidosis, hATTR or ATTR). RNAi is a cellular process of gene silencing. Patisiran (Onpattro) silences specific messenger RNA, blocking the production of transthyretin protein. This enables the clearance of TTR amyloid deposits from and restores function in peripheral tissues.

Inotersen (Tegsedi^TM) is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) protein synthesis indicated for the treatment of hereditary transthyretin amyloidosis (TTR amyloidosis, hATTR or ATTR). Inotersen (Tegsedi) causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Vutrisiran (Amvuttra™) is a transthyretin-directed small interfering RNA for the treatment of heredity transthyretin amyloidosis (TTR amyloidosis, hATTR or ATTR) associated polyneuropathy.

Treatment with patisiran (Onpattro), inotersen (Tegsedi), or vutrisiran (Amvuttra) typically leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance of vitamin A is advised for individuals. Higher doses than the recommended daily allowance should not be given to try to achieve normal serum vitamin A levels during treatment, as serum vitamin A levels do not reflect the total vitamin A in the body. Individuals should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (eg. night blindness).

Hereditary transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal deposits of amyloid protein in the body's organs and tissues. It is caused by mutations in the TTR gene. The TTR gene provides instructions for producing a protein called transthyretin, which transports vitamin A and thyroxine throughout the body. Transthyretin is produced primarily in the liver. A small amount is produced in the choroid plexus of the brain and in the retina. In TTR amyloidosis, transthyretin builds up as amyloid fibrils in tissues (primarily liver), interfering with the normal function of these tissues. The ideal setting for evaluation of the individual with hereditary transthyretin amyloidosis is a multi-disciplinary Amyloid Program.

Note: Onpattro is an intravenous infusion and may be covered under the medical benefit. Amvuttra is a healthcare administered subcutaneous injection and may be covered under the medical benefit. Tegsedi is a subcutaneous injection and may be covered under the pharmacy benefit.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

The use of patisiran (Onpattro) or vutrisiran (Amvuttra) may be considered medically necessary when ALL of the following criteria are met:

The individual must meet FDA-approved label for use (e.g., use outside of studied population will be considered investigational); and
The requested medication must be prescribed by, or in consult with, a neurologist, geneticist, or specialist in the treatment of amyloidosis; and
Documentation of genetic testing confirming a pathogenic TTR mutation (e.g., V30M) must be provided; and
Documentation of ONE of the following must be provided:
- Baseline polyneuropathy disability (PND) score less than or equal to IIIb; or
- Baseline FAB Stage 1 or 2; or
- Baseline neuropathy impairment (NIS) score greater than or equal to 10 and less than or equal to 130; and
The individual has not had a liver transplant; and
The individual has clinical signs and symptoms of the disease (amyloid deposition in biopsy specimens, TTR protein variants in serum, weakness, sensory loss, decreased motor strength, decreased gait speed, etc.); and
The individual is not receiving any other TTR reducing agent (i.e., vutrisiran, patisiran, tafamidis, inotersen).

Initial Authorization: Six (6) months

Reauthorization Criteria

Continuation of therapy with patisiran (Onpattro) and vutrisiran (Amvuttra) may be considered medically necessary when the following is met:

Documentation of a therapeutic response as evidenced by stabilization or improvement (e.g., improved neurologic impairment, motor function, quality of life, slowing of disease progression, etc.) from baseline in ONE of the following:
- Baseline polyneuropathy disability (PND) score less than or equal to IIIb; or
- Baseline FAB Stage 1 or 2; or
- Baseline neuropathy impairment (NIS) score greater than or equal to 10 and less than or equal to 130.

The use of patisiran (Onpattro) and vutrisiran (Amvuttra) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.

Procedure Codes

J0222

J0225

Tegsedi (inotersen) is a subcutaneous injection and may be covered under the pharmacy benefit.

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the U.S. Food and Drug Administration (FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines.

Diagnosis Codes

E85.1

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021- Effective January 01, 2022

Adopted Medicaid Expansion specific policy

Internal Medical Policy Committee 11-29-2022 - Effective January 1, 2023

Added vutrisiran (Amvuttra) to the policy, and
Updated initial criteria, and
Updated reauthorization criteria, and
Updated experimental/investigational statement

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving, and the Company reserves the right to review and update medical policy periodically.

Policy ID: ME-I-201-005-01

Section: Injections

Effective Date: January 01, 2022

Last Reviewed: November 23, 2021

Archived Date: December 31, 2022

Applies To: Medicaid Expansion Only

Description

Treatment with patisiran (Onpattro) or inotersen (Tegsedi) typically leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance of vitamin A is advised for individuals. Higher doses than the recommended daily allowance should not be given to try to achieve normal serum vitamin A levels during treatment, as serum vitamin A levels do not reflect the total vitamin A in the body. Individuals should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (eg. night blindness).

Note: Onpattro is an intravenous infusion and may be covered under the medical benefit. Tegsedi is a subcutaneous injection and may be covered under the pharmacy benefit.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

The use of patisiran (Onpattro) may be considered medically necessary when ALL of the following criteria are met:

The individual must meet criteria as outlined in prescribing information (PI) including recommendations for diagnosis and age; and
The prescriber is a, or in consult with, a neurologist, geneticist, or specialist in the treatment of amyloidosis; and
The individual must have a diagnosis of polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis (hATTR) confirmed by both of the following (as evidenced with submitted documentation):
- Any transthyretin (TTR) mutation confirmed by genetic testing; and
- Clinical signs and symptoms of the disease (e.g., peripheral/autonomic neuropathy, motor disability); and
The provider must submit documentation of one of the following:
- Neuropathy Impairment Score (NIS) of five (5) or greater; or
- Polyneuropathy disability (PND) score of ≤ IIIb; or
- Familial amyloid polyneuropathy (FAP) of stage 1 or 2.

Initial Authorization: 6 months

Reauthorization Criteria

Continuation of therapy with patisiran (Onpattro) may be considered medically necessary when the following is met:

The individual must have experienced meaningful clinical benefit since starting treatment with the requested medication, as evidenced by medical documentation (e.g. chart notes) attached to the request (subject to clinical review) including maintenance or improvement in the one of the following scores and symptoms:
- NIS score; or
- PND score; or
- FAP stage.

Continuation Authorization: 12 months

The use of patisiran (Onpattro) is considered experimental/investigational and, therefore, non-covered for all other indications. Scientific literature does not support its use for other indications.

Procedure Codes

J0222

Tegsedi (inotersen) is a subcutaneous injection and may be covered under the pharmacy benefit.

Diagnosis Codes

E85.1

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 11-23-2021 Adopted Medicaid Expansion specific policy effective 1-1-2022

Links

References (PDF)

Disclaimer

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Treatment of Hereditary Amyloidosis (Medicaid Expansion)

Description

Criteria

Diagnosis Codes

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer

Description

Criteria

Procedure Codes

Diagnosis Codes

Professional Statements and Societal Positions Guidelines

ND Committee Review

Links

Disclaimer