Pharmacy Prior Authorization Criteria

Symdeko (tezacaftor/ivacaftor)

Indications for Prior Authorization

Symdeko (tezacaftor/ivacaftor)

• For diagnosis of Cystic Fibrosis (CF)
  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 a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Criteria

Symdeko

*Please consult Background section for table of CFTR gene mutations responsive to Symdeko. For initial authorization request, approve through 12/31/2039 For reauthorization request, bypass criteria review and approve through 12/31/2039

Prior Authorization

Length of Approval: When approved; no reauthorization required

• Patient is 6 years of age or older
AND
• Diagnosis of cystic fibrosis (CF) [2,3]
AND
• One of the following:
• Patient is homozygous for the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene as detected by a U.S. Food and Drug Administration (FDA)-cleared cystic fibrosis mutation test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
OR
• Patient has at least one mutation in the CFTR gene that is responsive to tezacaftor/ivacaftor based in vitro data and/or clinical evidence* as detected by a U.S. Food and Drug Administration (FDA)-cleared cystic fibrosis mutation test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
AND
• Prescribed by or in consultation with one of the following:
• Pulmonologist
• Specialist affiliated with a CF care center

P & T Revisions

2025-04-30, 2025-04-30, 2025-03-25, 2025-02-04, 2024-02-01, 2023-11-30, 2023-01-09, 2022-02-03, 2021-02-04, 2020-04-01, 2019-07-30

References

1. Symdeko Prescribing information. Vertex Pharmaceuticals Inc. Boston, MA. August 2023.
2. Cousar-Taylor J, Munch A, McKone E, et al. Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for phe508del. N Engl J Med. 2017; 1-11.
3. Rowe S, Daines C, Ringshausen F, et al. Tezacaftor-ivacaftor in residual-function heterozygotes with cystic fibrosis. N Engl J Med. 2017; 1-12.

---

Revision History

• 2025-04-30: Approval length updated to "Approved- no reauthorization required"
• 2025-04-30: Note and approval updated from 2099 to 2039.
• 2025-03-25: Removing reauthorization requirement as part of extended reauthorization program.
• 2025-02-04: Annual review: No criteria changes.
• 2024-02-01: No criteria changes. Background and reference updates.
• 2023-11-30: Program update to standard reauthorization language. No changes to clinical intent.
• 2023-01-09: Annual review: No criteria changes.
• 2022-02-03: Annual review: no criteria changes
• 2021-02-04: 2021 Annual Review. Added background table and edited criteria to align with updated labeling including additional CFTR mutations.
• 2020-04-01: 2020 Annual Review; no changes to criteria
• 2019-07-30: 1) Modify PA due to expanded age: Treatment of patients with cystic fibrosis (CF) 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. 2) New formulation: tezacaftor 50mg/ivacaftor 75mg and 75 mg - add to updated guideline"

---