Selection of CFTR modulator therapy for patients with cystic fibrosis who are <2 years old

The above table shows mutations approved for Iva by the US Food and Drug Administration as of May 2023. Other options for CFTR modulator therapy will be available for most patients once they reach ≥2 years of age.

CF: cystic fibrosis; CFTR: cystic fibrosis transmembrane conductance regulator; ETI: elexacaftor-tezacaftor-ivacaftor (TRIKAFTA); Iva: ivacaftor (KALYDECO); Lum-Iva: lumacaftor-ivacaftor (ORKAMBI).

* For patients who are F508del homozygotes, we suggest Lum-Iva beginning at age 1 year and advancing to ETI at age 2 years.

¶ 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.

Data from:

KALYDECO (ivacaftor tablets and oral granules). Revised August 2023. US Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/203188s040,207925s017lbl.pdf (Accessed on January 14, 2025).

Graphic 130531 Version 8.0

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Selection of CFTR modulator therapy for patients with cystic fibrosis who are <2 years old