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. 2025 Mar;24(2):255-262.
doi: 10.1016/j.jcf.2024.07.015. Epub 2024 Jul 29.

Characteristics of individuals with cystic fibrosis in the United States ineligible for ivacaftor and elexacaftor/tezacaftor/ivacaftor

Don B Sanders  1 , Nicole Mayer-Hamblett  2 , Margaret Rosenfeld  3 , Deepika Polinieni  4 , Elliott Dasenbrook  5 , Rhonda Szczesniak  6 , Elizabeth A Cromwell  7
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Free article

Characteristics of individuals with cystic fibrosis in the United States ineligible for ivacaftor and elexacaftor/tezacaftor/ivacaftor

Don B Sanders et al. J Cyst Fibros. 2025 Mar.
Free article

Abstract

Background: We characterized people with cystic fibrosis (CF) ineligible by genotype (not age) for currently approved CFTR modulator therapy using data from the US CF Foundation Patient Registry (CFFPR).

Methods: We summarized clinical characteristics using CFFPR data from 2017 to 2022. Annual rate of change in percent predicted of forced expiratory volume in one second (ppFEV1) was estimated using generalized estimating equations.

Results: A total of 2,790 individuals with CF met inclusion criteria. In 2022, 12 % were less than 6 years old, 16 % were age 6-12 years, 18 % age 12-18 years and 54 % were ≥18 years. The proportion identified as White was 74 %, 17 % Black, and 26 % as Hispanic. The median (IQR) age at diagnosis was 1.2 (0.5, 9.1) months for children and 3.1 (0.3, 17.4) years for adults. Median (IQR) ppFEV1 among children was 91.9 (80.3; 102.4) and among adults, 74.3 (52.4; 90.4). Pancreatic enzymes were prescribed for 77.8 %. Population-level average (95 % CI) rates of decline in ppFEV1 among the pancreatic insufficient population was -1.5 per year (-1.8; -1.2) for ages 6 to <11 years, -2.2 per year (-2.6; -1.8) for ages 12 to <18 years, and -1.5 per year (-1.7; -1.3) for adults.

Conclusions: We describe the CFTR modulator ineligible population in the US in 2017-2022. With a growing pipeline of therapies aimed at improving CFTR function for those who cannot benefit from modulators due to ineligibility, characterization of both the size and outcomes of these populations are critical to inform optimal clinical development plans and future clinical trials.

Keywords: CFTR modulator ineligible; Cystic fibrosis.

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Conflict of interest statement

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Elizabeth Cromwell reports financial support was provided by Cystic Fibrosis Foundation. If there are other authors, they declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

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