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.

CFTR: cystic fibrosis transmembrane conductance regulator; CF: cystic fibrosis; IVA: ivacaftor (KALYDECO); LUM-IVA: lumacaftor-ivacaftor (ORKAMBI); ELX-TEZ-IVA: elexacaftor-tezacaftor-ivacaftor (TRIKAFTA).

* For patients who are F508del homozygotes, we suggest LUM-IVA for ages 1 to 2 years, then advancing to ELX-TEZ-IVA at age 2 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.

Data from:

ORKAMBI® (lumacaftor/ivacaftor) Tablets and Oral Granules. Vertex Pharmaceuticals Incorporated. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/211358s004lbl.pdf (Accessed on September 9, 2022).
KALYDECO® (ivacaftor) Tablets and Oral Granules. Vertex Pharmaceuticals Incorporated. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/207925s016lbl.pdf (Accessed on May 9, 2023).

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