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FDA Broadens CFTR Therapy Eligibility

The FDA expanded use of CFTR modulators to include more CF gene variants, increasing treatment eligibility across a wider US patient population.

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  • 1

    FDA approved expanded indications for CFTR modulators.

  • 2

    Approximately 95% of cystic fibrosis patients are now eligible.

  • 3

    Vanzacaftor/tezacaftor/ivacaftor for ages 6+.

  • 4

    Elexacaftor/tezacaftor/ivacaftor for ages 2+.

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    800 additional patients eligible after expansion.

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    Monitor liver function regularly for patients on these treatments.

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    Common side effects include cough and upper respiratory infections.

Original Source(s)

FDA Broadens CFTR Therapy Eligibility

  • by Kathryn Wighton

  • April 1, 2026

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