Subject: Kalydeco 1884-A SGM P2024
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 Incorporated; August 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. doi: 10.1542/peds.2015-1784
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 09, 2024
