Subject: Vyndaqel-Vyndamax 3076-A SGM P2022

Drug

VYNDAGEL  (tafamidis meglumine)

VYNDAMAX  (tafamidis)

Policy:

I.    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 Indication
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.

II.  DOCUMENTATION 

Submission of the following information is necessary to initiate the prior authorization review:
A.   Echocardiography or cardiac magnetic resonance imaging results confirming cardiac involvement
B.   For members with hereditary ATTR-CM: results confirming a mutation of the transthyretin (TTR) gene
C.   For biopsy proven disease:
      1.   Tissue biopsy confirming the presence of the transthyretin amyloid deposition
      2.   Immunohistochemical analysis, mass spectrometry, tissue staining, or polarized light microscopy
            results confirming transthyretin precursor proteins
D.   For technetium-labeled bone scintigraphy proven disease:
      1.   A serum kappa/lambda free light chain ratio, serum protein immunofixation or urine protein
            immunofixation test result showing the absence of monoclonal proteins
      2.   Scintigraphy tracing results confirming presence of amyloid deposits
E.   For continuation of therapy: 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, NYHA classification of heart
      failure, left ventricular stroke volume, NT-proBNP level)

III. CRITERIA FOR INITIAL APPROVAL 

Cardiomyopathy of Wild Type or Hereditary Transthyretin-mediated Amyloidosis
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:
A.   The member exhibits clinical symptoms of cardiomyopathy and heart failure (e.g., dyspnea, fatigue,
      orthostatic hypotension, syncope, peripheral edema).
B.   Cardiac involvement was confirmed by echocardiography or cardiac magnetic resonance imaging
      (e.g., end-diastolic interventricular septal wall thickness exceeding 12 mm).
C.   The diagnosis is confirmed by one of the following:
      1.   The member meets both of the following:
            i.    Presence of transthyretin amyloid deposits on analysis of biopsy from cardiac or noncardiac sites.
            ii.   Presence of transthyretin precursor proteins was confirmed by immunohistochemical analysis,
                  mass spectrometry, tissue staining, or polarized light microscopy.
      2.   The member meets both of the following:
            i.    Positive technetium-labeled bone scintigraphy tracing.
            ii.   Systemic light chain amyloidosis is ruled out by a test showing absence of monoclonal
                  proteins (serum kappa/lambda free light chain ratio, serum protein immunofixation, or
                  urine protein immunofixation).
D.   For members with hereditary ATTR-CM, presence of a mutation of the TTR gene was confirmed.
E.   The member is not a liver transplant recipient.
F.   The requested medication will not be used in combination with inotersen (Tegsedi) or patisiran (Onpattro).

IV.  CONTINUATION OF THERAPY 

Authorization of 12 months may be granted for the continued treatment of ATTR-CM when all of the following criteria are met:
A.   The member must meet all initial authorization criteria.
B.   The 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]. Documentation from the medical
      record must be provided.

Place of Service:

Outpatient

The above policy is based on the following references:

1. Vyndaqel and Vydamax [package insert]. New York, NY: Pfizer Labs.; June 2021.
2. Maurer MS, Schwartz JH, Gundapaneni B, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018 Sep 13; 379(11):1007-1016.
3. Clinical Consult. CVS Caremark Clinical Programs Review: Focus on Heart Failure (HF) Clinical Programs. April 2020.
4. Mauer MS, Sabahat B, Thibaud D, et al. Expert Consensus Recommendations for the Suspicion and Diagnosis of Transthyretin Cardiac Amyloidosis. Circulation: Heart Failure. 2019 Sep 4;12:9.

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.

August 14, 2022