Pharmacy Prior Authorization Criteria

Kalydeco (ivacaftor)

For diagnosis of Cystic fibrosis
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 a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

*Please consult Background section for table of CFTR gene mutations responsive to Kalydeco.

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

AND

Patient has 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* as detected by an FDA-cleared cystic fibrosis mutation test or a test performed at a Clinical Laboratory Improvement Amendments (CLIA)-approved facility

AND

AND

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

Patient demonstrates positive clinical response (i.e., improvement in lung function [percent predicted forced expiratory volume in one second {PPFEV1}], decreased number of pulmonary exacerbations) to therapy [A]

2024-02-01, 2023-11-23, 2023-10-03, 2023-06-27, 2023-01-06, 2022-02-03, 2021-09-27, 2021-05-19, 2021-02-04, 2020-11-03, 2020-04-01

Kalydeco Prescribing Information. Vertex Pharmaceuticals Incorporated. Boston, MA. August 2023.
Ramsey BW, Davies J, McElvaney G, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663-1672.

The primary efficacy endpoint in both Kalydeco pivotal trials was improvement in lung function as determined by the mean absolute change from baseline in percent predicted pre-dose FEV1 through 24 weeks of treatment. [2]

2024-02-01: No criteria changes. Updated background and references.
2023-11-23: Addition of Kalydeco 5.8mg granules as target.
2023-10-03: Program update to standard reauthorization language. No changes to clinical intent
2023-06-27: Addition of Kalydeco 13.6mg granules and update age criteria to 1 month of age or older based on new indication
2023-01-06: Annual review: No criteria changes.
2022-02-03: Annual review: no criteria changes
2021-09-27: Addition of EHB formulary to guideline, no changes to criteria
2021-05-19: Addition of EHB formulary to guideline, no changes to criteria
2021-02-04: 2021 Annual Review. Added background table and edited criteria to align with updated labeling including additional CFTR mutations.
2020-11-03: Updated criteria due to expanded age range of 4 months and older.
2020-04-01: 2020 Annual review; no changes to criteria