Orkambi (lumacaftor/ivacaftor) — Medica

Cystic fibrosis, homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene

Initial criteria

age ≥ 1 year
Patient has TWO copies of the F508del mutation in the CFTR gene
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 signs and symptoms of cystic fibrosis (examples include meconium ileus, sino-pulmonary symptoms, bronchiectasis, sinusitis, failure to thrive, pancreatic insufficiency)
Patient has evidence of abnormal CFTR function 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

1 year