Subject: Symdeko 2516-A SGM P2023

Drug

SYMDEKO  (tezacaftor/ivacaftor)

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
          Symdeko is indicated for the treatment of cystic fibrosis (CF) in
          patients age 6 years and older who are homozygous for the
          F508del mutation or who have at least one mutation in the
          cystic fibrosis transmembrane conductance regulator (CFTR)
          gene that is responsive to tezacaftor/ivacaftor based on in vitro
          data and/or clinical evidence.

          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.

    II.  PRESCRIBER SPECIALTIES

          This medication must be prescribed by or in consultation with a
          pulmonologist.

 
  III.  CRITERIA FOR INITIAL APPROVAL

          Cystic Fibrosis
          Authorization of 12 months may be granted for treatment of
          cystic fibrosis when all of the following criteria are met:
          A.  Genetic testing was conducted to detect a mutation in the
               CFTR
          B.  The member is homozygous for the F508del mutation, or the
               member has one of the following mutations in the CFTR
               gene: A120T, A234D, A349V, A455E, A554E, A1006E,
               A1067T, D110E, D110H, D192G, D443Y,
               D443Y;G576A;R668C, D579G, D614G, D836Y, D924N,
               D979V, D1152H, D1270N, E56K, E60K, E92K, E116K, E193K,
               E403D, E588V, E822K, E831X, F191V, F311del, F311L,
               F508C, F508C;S1251N, F575Y, F1016S, F1052V, F1074L,
               F1099L, G126D, G178E, G178R, G194R, G194V, G314E,
               G551D, G551S, G576A, G576A;R668C, G622D, G970D,
               G1069R, G1244E, G1249R, G1349D, H939R, H1054D,
               H1375P, I148T, I175V, I336K, I601F, I618T, I807M, I980K,
               I1027T, I1139V, I1269N, I1366N, K1060T, L15P, L206W,
               L320V, L346P, L967S, L997F, L1324P, L1335P, L1480P,
               M152V, M265R, M952I, M952T, P5L, P67L, P205S, Q98R,
               Q237E, Q237H, Q359R, Q1291R, R31L, R74Q, R74W,
               R74W;D1270N, R74W;V201M, R74W;V201M;D1270N, R75Q,
               R117C, R117G, R117H, R117L, R117P, R170H, R258G,
               R334L, R334Q, R347H, R347L, R347P, R352Q, R352W,
               R553Q, R668C, R751L, R792G, R933G, R1066H, R1070Q,
               R1070W, R1162L, R1283M, R1283S, S549N, S549R, S589N,
               S737F, S912L, S945L, S977F, S1159F, S1159P, S1251N,
               S1255P, T338I, T1036N, T1053I, V201M, V232D, V562I,
               V754M, V1153E, V1240G, V1293G, W1282R, Y109N, Y161S,
               Y1014C, Y1032C, 546insCTA, 711+3A→G, 2789+5G→A,
               3272-26A→G, 3849+10kbC→T.
          C.  The member is at least 6 years of age.
          D.  Symdeko 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:

1. Symdeko [package insert]. Boston, MA: Vertex Pharmaceuticals Inc.; June 2022.
2. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-Ivacaftor in Residual Function Heterozygotes with Cystic Fibrosi N Engl J Med. 2017;377:2024-2035.
3. Taylor-Cousar JL, Munck A, McKone EF, et al. Tezacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017;377:2013-2023.

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