Kalydeco (ivacaftor) — Medica

Initial criteria

• Patient is age ≥ 1 month
• Patient has at least ONE pathogenic or likely pathogenic CFTR mutation responsive to Kalydeco (E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, D1270N, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, 2789+5G—>A, 3272-26A—>G, 3849+10kbC—>T, 711+3A—>G, E831X, R117H, A120T, A234D, A349V, D192G, D924N, E882K, F311L, F311del, F508C, F508C;S1251N, G178E, G194R, G314E, G576A, G970D, G1249R, H939R, H1375P, I148T, I175V, I807M, I1027T, I1139V, L320V, L967S, L997F, L1480P, M152V, M9521, M952T, Q237E, Q237H, Q359R, Q1291R, R75Q, R117G, R117L, R117P, R170H, R347L, R553Q, R668C, R792G, R933G, R1162L, R1283M, S589N, S737F, S1159F, S1159P, T338I, T1053I, V232D, V562I, V754M, V1293G, W1282R, Y1014C, Y1032C)
• Patient meets at least ONE of the following: positive cystic fibrosis newborn screening test OR family history of cystic fibrosis OR clinical presentation consistent with cystic fibrosis (e.g., meconium ileus, persistent cough, wheezing, bronchiectasis, sinusitis, failure to thrive, pancreatic insufficiency)
• Patient has evidence of abnormal CFTR function as demonstrated by at least ONE of the following: elevated sweat chloride test OR two cystic fibrosis–causing CFTR mutations OR abnormal nasal potential difference
• Medication is prescribed by or in consultation with a pulmonologist or a physician who specializes in the treatment of cystic fibrosis

Approval duration

1 year