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Related Concept Videos

Cystic Fibrosis: Management01:24

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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
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The concept of therapeutic equivalence (TE) in drugs with multiple indications is complex. A generic drug may be therapeutically equivalent to a brand-name product for one specific indication, but this doesn't necessarily mean it's equivalent for all other indications. Evidence of TE in one patient group and bioequivalence shown in healthy volunteers can support—but not confirm—TE for other indications. However, definitive proof requires individual clinical studies for each...
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Related Experiment Video

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Rethinking CFTR variant responsiveness: Differential responses to vanzacaftor and elexacaftor.

Noelia Rodriguez Mier1, Isabelle Callebaut2, Marijke Proesmans1

  • 1Department of Development and Regeneration, Woman and Child Unit, CF research lab, KU Leuven, Leuven, Belgium; Department of Paediatrics, Paediatric Pulmonology, University Hospital Leuven, Leuven, Belgium.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|January 1, 2026
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Summary

New cystic fibrosis (CF) modulator vanzacaftor/tezacaftor/deutivacaftor (VTD) shows promise for patients with previously unresponsive CFTR variants. VTD offers improved efficacy and potential benefits where other treatments failed, advancing personalized CF care.

Keywords:
CFTR modulatorsCystic fibrosisElexacaftorIn vitro functional assaysPatientderived intestinal organoidsPersonalized therapyRare CFTR variantsTheratypingVanzacaftor

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Area of Science:

  • Pulmonology and Genetics
  • Drug Discovery and Development

Background:

  • Cystic fibrosis (CF) treatments have advanced with CFTR modulators like elexacaftor/tezacaftor/ivacaftor (ETI).
  • A significant number of individuals with CF (pwCF) carry CFTR variants unresponsive to ETI, necessitating novel therapeutic strategies.
  • Next-generation modulators are crucial for addressing unmet needs in CF treatment.

Purpose of the Study:

  • To evaluate the efficacy of the next-generation modulator combination vanzacaftor/tezacaftor/deutivacaftor (VTD).
  • To assess VTD's potential benefit for CFTR variants previously classified as ETI-unresponsive.
  • To explore the distinct mechanisms of action between VTD and ETI based on variant-specific responses.

Main Methods:

  • Analysis of Phase 3 trial data for VTD efficacy, focusing on sweat chloride reduction.
  • Reassessment of 31 CFTR variants previously deemed ETI-unresponsive using updated in vitro data and molecular interaction insights.
  • Utilizing patient-derived intestinal organoid (PDIO) assays to compare VTD and ETI functional responses in specific CFTR mutations (G458V, G85R).

Main Results:

  • Phase 3 trials indicate superior efficacy of VTD compared to ETI in reducing sweat chloride levels.
  • Reassessment suggests some previously ETI-unresponsive variants exhibit residual function or borderline responsiveness.
  • PDIO assays demonstrated significant functional improvement with VTD, but not ETI, in specific patient mutations, supporting different mechanisms of action.

Conclusions:

  • VTD demonstrates significant therapeutic potential for pwCF with rare or previously ETI-unresponsive CFTR variants.
  • Current in vitro thresholds for classifying CFTR variant responsiveness may need refinement.
  • Personalized, data-driven approaches incorporating advanced assays like PDIOs are essential for optimizing CF care and treatment decisions.