CFTR: cystic fibrosis transmembrane conductance regulator; CF: cystic fibrosis; IVA: ivacaftor (KALYDECO); LUM-IVA: lumacaftor-ivacaftor (ORKAMBI).
* For patients who are F508del homozygotes, we suggest LUM-IVA for ages 1 to 5 years, then advancing to ELX-TEZ-IVA at age 6 years. F508del homozygotes are also eligible for TEZ-IVA, but the drug is not approved for children less than 6 years old.
¶ For patients who are not eligible for any available CFTR modulator, consider enrollment in a clinical trial, if available for the patient's genotype and age.