Subject: Vyndaqel-Vyndamax 3076-A SGM P2023
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VYNDAQEL
(tafamidis meglumine)
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VYNDAMAX
(tafamidis)
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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), patisiran (Onpattro), or vutrisiran
(Amvuttra).
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:
- Vyndaqel and Vyndamax [package insert]. New York, NY: Pfizer Labs; April 2023.
- 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.
- Maurer MS, Sabahat B, Thibaud D, et al. Expert consensus recommendations for the suspicion and diagnosis of transthyretin cardiac amyloidosis. Circ Heart Fail. 2019 Sep 4;12:9.
- Ruberg FL, Grogan M, et al. Transthyretin amyloid cardiomyopathy. J Am Coll Cardiol. 2019;73:2872-91.
- Yadav JD, Othee H, Chan KA, Man DC, Belliveau PP, Towle J. Transthyretin Amyloid Cardiomyopathy-Current and Future Therapies. Ann Pharmacother. 2021;55(12):1502-1514.
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.
October 14, 2023
