CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Medicaid_Vyndaqel Vyndamax.20250514

Covers Vyndaqel and Vyndamax for treatment of cardiomyopathy of wild-type or variant/hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults when specified clinical and diagnostic criteria are met; all other indications are experimental/investigational and not medically necessary. Policy applies to Medicaid members.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMedicaid_Vyndaqel Vyndamax.20250514

Policy CodePolicy N/A

Change TypeClarified (reviewed; no material change)

Effective DateApr 1, 2020

Next Review Date

Key ActionPrior authorization required; authorization may be granted for 6 months for initial therapy and 6 months for continuation when all listed criteria are documented.

SourceLink

POLICY UPDATE CHANGES

Policy retained effective date 4/1/2020 and lists reviews through 5/2025 with scope: Medicaid.

2FDA-approved indications covered

6Initial authorization (months)

6Continuation authorization (months)

V20:4/V61:1Quantity limits

Scope: Covers Vyndaqel and Vyndamax (tafamidis) for treatment of cardiomyopathy of wild-type or variant/hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults when the specified clinical and diagnostic criteria are met; all other indications are considered experimental/investigational and not medically necessary. Policy applies to Medicaid members. Effective date: 4/1/2020; Last review: 5/2025.

Key thresholds/limits

Echocardiography/CMR wall thicknessEnd-diastolic interventricular septal wall thickness exceeding 12 mm

NYHA class allowedClass I, II, or III (not IV)

Continuation requirement — 6MWTImprovement in distance walked on the 6-minute walk test

Continuation requirement — KCCQ-OSImprovement in Kansas City Cardiomyopathy Questionnaire - Overall Summary score

Continuation requirement — CV hospitalizationsReduction in cardiovascular-related hospitalizations

Continuation requirement — LV stroke volumeImproved left ventricular stroke volume

Continuation requirement — NT-proBNPChange/improvement in N-terminal B-type natriuretic peptide (NT-proBNP) level

Initial Authorization (up to 6 months) - ATTR-CM

Authorization of 6 months may be granted when ALL of the following criteria are met:

ALL of the following

Vyndaqel or Vyndamax is prescribed by, or in consultation with, a cardiologist
Documentation that the member exhibits clinical symptoms of cardiomyopathy and heart failure classified as New York Heart Association (NYHA) class I, II, or III diseaseNYHA class I-III
Examples: dyspnea, fatigue, orthostatic hypotension, syncope, peripheral edema
Documentation that cardiac involvement was confirmed by echocardiography or cardiac magnetic resonance imaging
Example: end-diastolic interventricular septal wall thickness exceeding 12 mm
Diagnosis confirmation (ONE of the following two pathways):
- Pathway 1 - Tissue biopsy confirmation
  - Presence of transthyretin amyloid deposits on analysis of biopsy from cardiac or noncardiac sites
  - Presence of transthyretin precursor proteins confirmed by immunohistochemical analysis, mass spectrometry, tissue staining, or polarized light microscopy
- Pathway 2 - Scintigraphy plus exclusion of light-chain disease
  - Positive technetium-labeled bone scintigraphy tracing
  - Systemic light chain amyloidosis is ruled out by tests showing absence of monoclonal proteins (serum kappa/lambda free light chain ratio, serum protein immunofixation, or urine protein immunofixation)
For variant/hereditary ATTR-CM: documentation of presence of a mutation of the TTR gene
The member is not a liver transplant recipient
The requested medication will not be used in combination with acoramidis (Attruby), patisiran (Onpattro), eplontersen (Wainua), or vutrisiran (Amvuttra)

Special Combination-Use Clause

Special combination-use clause

If combination therapy is requested for overlapping ATTR-CM and ATTR-PN:

If request is for Vyndaqel/Vyndamax in combination with a TTR-silencer/reducer (e.g., patisiran, eplontersen) to treat overlapping ATTR-CM and ATTR-PN, the member must have a documented inadequate response, intolerance, or contraindication to monotherapy vutrisiran (Amvuttra)

Denial Risk

Combination therapy restriction

Requests to combine tafamidis (Vyndaqel or Vyndamax) with acoramidis (Attruby), patisiran (Onpattro), eplontersen (Wainua), or vutrisiran (Amvuttra) are not permitted unless the policy's specific combination-use exception is met. For overlapping ATTR-CM and ATTR-PN, combination with a TTR-silencer/reducer (e.g., patisiran, eplontersen) is allowed only when the member has a documented inadequate response, intolerance, or contraindication to monotherapy vutrisiran (Amvuttra).

Continuation of Therapy (up to 6 months)

Authorization of 6 months may be granted for continued treatment when ALL of the following criteria are met:

ALL of the following

Documentation that the member meets all initial authorization criteria
Documentation that the member has demonstrated a beneficial response to tafamidis therapy
Examples of measures: improvement in rate of disease progression as demonstrated by 6-minute walk test distance, Kansas City Cardiomyopathy Questionnaire - Overall Summary (KCCQ-OS) score, cardiovascular-related hospitalizations, left ventricular stroke volume, N-terminal B-type natriuretic peptide (NT-proBNP) level
Documentation that the member has not progressed to NYHA class IV diseaseNYHA not IV

Provider Actions & Billing Rules

Prior Authorization

Authorization requirement

Prior authorization is required. Authorization may be granted for up to 6 months for initial therapy and up to 6 months for continuation when all applicable criteria are documented.

Documentation Required

Diagnostic confirmation documentation

Diagnosis must be confirmed by ONE of the following pathways: (1) biopsy showing transthyretin amyloid deposits plus confirmation of transthyretin precursor proteins (immunohistochemistry, mass spectrometry, tissue staining, or polarized light microscopy); OR (2) positive technetium-labeled bone scintigraphy tracing plus exclusion of systemic light-chain amyloidosis by demonstrating absence of monoclonal proteins using serum kappa/lambda free light chain ratio and serum or urine protein immunofixation.

Biopsy pathology showing transthyretin amyloid deposits
Confirmation of transthyretin precursor proteins by immunohistochemical analysis, mass spectrometry, tissue staining, or polarized light microscopy
Technetium-labeled bone scintigraphy (positive)
Serum kappa/lambda free light chain ratio (to rule out monoclonal protein)
Serum protein immunofixation and/or urine protein immunofixation (to rule out monoclonal protein)

Documentation Required

Prescriber specialty documentation

Ensure the prescription is written by a cardiologist or that a cardiologist has been consulted and documented in the medical record.

Documentation Required

Genetic testing for variant ATTR

For variant/hereditary ATTR-CM, provide documentation confirming the presence of a pathogenic TTR gene mutation.

Billing Rule

Quantity limits

Quantity limits: Vyndaqel 20 mg — limit 4 capsules per day. Vyndamax 61 mg — limit 1 capsule per day.

Denial Risk

Combination therapy restriction

Tafamidis must not be used in combination with acoramidis, patisiran, eplontersen, or vutrisiran unless the policy's combination-use criteria are satisfied. Specifically, for requests to treat overlapping ATTR-CM and ATTR-PN with tafamidis plus a TTR-silencer/reducer, the member must have a documented inadequate response, intolerance, or contraindication to monotherapy vutrisiran (Amvuttra).

Background & Definitions

Tafamidis (Vyndaqel and Vyndamax) are transthyretin stabilizers indicated to reduce cardiovascular mortality and cardiovascular-related hospitalization in adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Non‑FDA indications are considered experimental/investigational and not medically necessary per this policy. This policy and its coverage criteria apply to Medicaid members.

ATTR-CM: Transthyretin-mediated amyloid cardiomyopathy (cardiac involvement of transthyretin amyloidosis).

NYHA class: New York Heart Association functional classification for heart failure (I-IV).

Revision History

2020-04-01effective_date

Policy effective date retained at 4/1/2020 (document lists effective date as 4/1/2020).

2020-01review

Document shows a review in 1/2020; multiple subsequent reviews are listed but no substantive policy change is stated.

2021-01review

Document shows a review in 1/2021; multiple subsequent reviews are listed but no substantive policy change is stated.

2022-01review

Document shows a review in 01/2022; multiple subsequent reviews are listed but no substantive policy change is stated.

2023-03review

Document shows a review in 3/2023; multiple subsequent reviews are listed but no substantive policy change is stated.

2024-03review

Document shows a review in 3/2024; multiple subsequent reviews are listed but no substantive policy change is stated.

2025-01review

Document shows a review in 1/2025; multiple subsequent reviews are listed but no substantive policy change is stated.

2025-05reviewLatest

Document shows a review in 5/2025 (latest listed review); multiple review dates are shown but the document indicates the policy was retained with no statement of substantive policy change. Scope: Medicaid.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyMedicaid_Vyndaqel Vyndamax.20250514

Policy CodePolicy N/A

Change TypeClarified (reviewed; no material change)

Effective DateApr 1, 2020

Next Review Date

Key ActionPrior authorization required; authorization may be granted for 6 months for initial therapy and 6 months for continuation when all listed criteria are documented.

SourceLink

On This Page

OpenPayer

Medicaid_Vyndaqel Vyndamax.20250514

Coverage Summary

Initial Authorization (up to 6 months) - ATTR-CM

Special Combination-Use Clause

Continuation of Therapy (up to 6 months)

Provider Actions & Billing Rules

Applicable Codes

Clinical Evidence & References

Background & Definitions

Revision History