Subject: Kalydeco 1884-A SGM P2023a
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
Kalydeco is indicated for the treatment of cystic fibrosis (CF) in
patients age 1 month and older who have at least one mutation
in the cystic fibrosis transmembrane conductance regulator
(CFTR) gene that is responsive to ivacaftor potentiation based
on clinical and/or in vitro assay data.
If the patient’s genotype is unknown, an FDA-cleared CF
mutation test should be used to detect the presence of CFTR
mutation followed by verification with bi-directional sequencing
when recommended by the mutation test instructions for use.
All other indications are considered experimental/investigational
and are not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate
the prior authorization review: For initial requests, genetic
testing report confirming the presence of the appropriate CFTR
gene mutation.
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a
pulmonologist.
IV. CRITERIA FOR INITIAL APPROVAL
Cystic Fibrosis
Authorization of 12 months may be granted for treatment of
cystic fibrosis when all the following criteria are met:
A. Genetic testing was conducted to detect a mutation in the
CFTR
B. The member has one of the following mutations in the CFTR
gene: A120T, A234D, A349V, A455E, A1067T, D110E, D110H,
D192G, D579G, D924N, D1152H, D1270N, E56K, E193K,
E822K, E831X, F311del, F311L, F508C, F508C;S1251N,
F1052V, F1074L, G178E, G178R, G194R, G314E, G551D,
G551S, G576A, G970D, G1069R, G1244E, G1249R, G1349D,
H939R, H1375P, I148T, I175V, I807M, I1027T, I1139V,
K1060T, L206W, L320V, L967S, L997F, L1480P, M152V,
M952I, M952T, P67L, Q237E, Q237H, Q359R, Q1291R,
R74W, R75Q, R117C, R117G, R117H, R117L, R117P, R170H,
R347H, R347L, R352Q, R553Q, R668C, R792G, R933G,
R1070Q, R1070W, R1162L, R1283M, S549N, S549R, S589N,
S737F, S945L, S977F, S1159F, S1159P, S1251N, S1255P,
T338I, T1053I, V232D, V562I, V754M, V1293G, W1282R,
Y1014C, Y1032C, 711+3A→G, 2789+5G→A, 3272-26A→G,
3849+10kbC→T.
C. The member is at least 1 month of age.
D. Kalydeco will not be used in combination with other
medications containing ivacaftor.
V. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization for an
indication listed in Section IV who are experiencing benefit from
therapy as evidenced by disease stability or disease
improvement (e.g., improvement in FEV1 from baseline).
Place of Service:
Outpatient
The above policy is based on the following references:
- Kalydeco [package insert]. Boston, MA: Vertex Pharmaceuticals Inc.; May 2023.
- Mogayzel PJ, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187:680-689.
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.
June 11, 2023
