Subject: Vyndaqel-Vyndamax SGM 3076-A P2025

Drug

VYNDAQEL  (tafamidis meglumine)

VYNDAMAX  (tafamidis)

Policy:

Indications

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-approved Indication¹

Vyndaqel and Vyndamax are transthyretin stabilizers indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

All other indications are considered experimental/investigational and not medically necessary.

Documentation

Submission of the following information is necessary to initiate the prior authorization review:

Initial requests

• Chart notes or medical record documentation of prior hospitalization of heart failure or confirming the member demonstrates clinical symptoms of heart failure at baseline.
• For biopsy proven disease:
  • Tissue biopsy from cardiac or noncardiac sites confirming the presence of the transthyretin amyloid deposition.
  • Immunohistochemical analysis, mass spectrometry, tissue staining, or polarized light microscopy results confirming the presence transthyretin precursor proteins.
• For technetium-labeled bone scintigraphy proven disease:
  • Scintigraphy tracing results confirming the presence of amyloid deposits.
  • Serum kappa/lambda free light chain ratio, serum protein immunofixation, and urine protein immunofixation test results showing the absence of monoclonal proteins.
• For hereditary ATTR-CM: testing or analysis confirming a pathogenic or likely pathogenic variant in the transthyretin (TTR) gene.

Continuation requests

Chart notes or medical record documentation confirming the member demonstrates a beneficial response to treatment (e.g., improvement in rate of disease progression as demonstrated by distance walked on the 6-minute walk test, the Kansas City Cardiomyopathy Questionnaire–Overall Summary [KCCQ-OS] score, cardiovascular-related hospitalizations, New York Heart Association [NYHA] classification of heart failure, left ventricular stroke volume, N-terminal B-type natriuretic peptide [NT-proBNP] level).

Prescriber Specialties

This medication must be prescribed by or in consultation with a geneticist, cardiologist, or a physician specializing in the treatment of amyloidosis.

Coverage Criteria

Cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM)^1-6

Authorization of 12 months may be granted for treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) when all of the following criteria are met:

• Member is 18 years of age or older.
• Member has medical history of heart failure with at least one prior hospitalization for heart failure (not due to arrhythmia or a conduction system disturbance treated with a permanent pacemaker), OR exhibits clinical symptoms of heart failure (e.g., volume overload, dyspnea, fatigue, orthostatic hypotension, syncope, peripheral edema) at baseline.
• Diagnosis is confirmed by either of the following criteria:
• Member meets both of the following criteria for biopsy proven disease:
  • Presence of transthyretin amyloid deposits on analysis of biopsy from cardiac or noncardiac sites.
  • Presence of transthyretin precursor proteins was confirmed by immunohistochemical analysis, mass spectrometry, tissue staining, or polarized light microscopy.
• Member meets both of the following criteria for technetium-labeled bone scintigraphy proven disease:
  • Presence of amyloid deposits confirmed by technetium-labeled bone scintigraphy tracing.
  • Systemic light chain amyloidosis is ruled out by showing the absence of monoclonal proteins by all of the following tests: a) serum kappa/lambda free light chain ratio, b) serum protein immunofixation, and c) urine protein immunofixation.
• For members with hereditary ATTR-CM, the diagnosis is confirmed by detection of a pathogenic or likely pathogenic variant in the TTR gene.
• Member does not have either of the following:
• A history of liver or heart transplant
• Implantation of left-ventricular assist device
• The requested medication will not be used in combination with inotersen (Tegsedi), patisiran (Onpattro), vutrisiran (Amvuttra), eplontersen (Wainua), or acoramidis (Attruby).

Continuation of Therapy

Authorization of 12 months may be granted for continued treatment of ATTR-CM when both of the following criteria are met:

• Member must meet all requirements in the coverage criteria.
• Member must have demonstrated a beneficial response to treatment with tafamidis therapy (e.g., improvement in rate of disease progression as demonstrated by distance walked on the 6-minute walk test, the Kansas City Cardiomyopathy Questionnaire–Overall Summary [KCCQ-OS] score, cardiovascular-related hospitalizations, NYHA classification of heart failure, left ventricular stroke volume, N-terminal B-type natriuretic peptide [NT-proBNP] level).

Place of Service:

Outpatient

The above policy is based on the following references:

1. Vyndaqel and Vyndamax [package insert]. New York, NY: Pfizer Labs; October 2023.
2. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379(11):1007-1016.
3. Maurer MS, Sabahat B, Thibaud D, et al. Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis. Circ Heart Fail. 2019;12(9):e006075.
4. Kittleson MM, Maurer MS, Ambardekar AV, et al. Cardiac amyloidosis: Evolving diagnosis and management: A scientific statement from the American Heart Association. Circulation. 2020;142(1):e7-e22.
5. Yadav JD, Othee H, Chan KA, et al. Transthyretin amyloid cardiomyopathy-Current and future therapies. Ann Pharmacother. 2021;55(12):1502-1514.
6. Kittleson MM, Ruberg FL, Ambardekar AV, et al. 2023 ACC expert consensus decision pathway on comprehensive multidisciplinary care for the patient with cardiac amyloidosis: A report of the American College of Cardiology solution set oversight committee. 2023;88(11):1076-1126.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

November 01, 2025