CFTR: cystic fibrosis transmembrane conductance regulator; ELX-TEZ-IVA: elexacaftor-tezacaftor-ivacaftor (TRIKAFTA); TEZ-IVA: tezacaftor-ivacaftor (SYMDEKO); IVA: ivacaftor (KALYDECO).
* For patients who are not eligible for any available CFTR modulator, consider enrollment in a clinical trial, if available for the patient's genotype.
¶ ELX-TEZ-IVA has not been approved for use in these 5 "splice site" mutations, because the assay used as a predictor of efficacy is not valid for this type of mutation. Clinical studies of ELX-TEZ-IVA have not included sufficient numbers of patients with these rare mutations to assess efficacy in this population. However, the demonstrated efficacy of IVA and TEZ-IVA for patients with these mutations suggests that ELX-TEZ-IVA would also be effective.Do you want to add Medilib to your home screen?