Science and Research

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3 Open-Label Extension Study

Rationale: Clinical and real-world studies show that elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) is efficacious and safe in people with cystic fibrosis (CF)

  • Daines, C. L.
  • Polineni, D.
  • Tullis, E.
  • Costa, S.
  • Linnemann, R. W.
  • Mall, M. A.
  • McKone, E. F.
  • Quon, B. S.
  • Ringshausen, F. C.
  • Selvadurai, H.
  • Taylor-Cousar, J. L.
  • Withers, N. J.
  • Sawicki, G. S.
  • Lee, T.
  • Ahluwalia, N.
  • Morlando Geiger, J.
  • Jennings, M.
  • Tan, Y. V.
  • Waltz, D.
  • Ramsey, B.
  • Griese, M.

Keywords

  • Humans
  • *Cystic Fibrosis/drug therapy/genetics
  • Male
  • *Quinolones/therapeutic use/adverse effects/administration & dosage
  • *Aminophenols/therapeutic use/adverse effects/administration & dosage
  • Female
  • Adult
  • Adolescent
  • *Benzodioxoles/therapeutic use/adverse effects/administration & dosage
  • *Indoles/therapeutic use/adverse effects/administration & dosage
  • Drug Combinations
  • *Cystic Fibrosis Transmembrane Conductance Regulator/genetics
  • Young Adult
  • Treatment Outcome
  • Child
  • *Pyrazoles/therapeutic use/adverse effects
  • *Pyrrolidines/therapeutic use/adverse effects
  • *Pyridines/therapeutic use/adverse effects
  • Chloride Channel Agonists/therapeutic use
  • Alleles
  • Middle Aged
  • Genotype
  • *Quinoxalines/therapeutic use/adverse effects
  • Quinolines
  • cystic fibrosis
  • elexacaftor
  • ivacaftor
  • long-term
  • tezacaftor
Publication details
DOI: 10.1164/rccm.202411-2231OC
Journal: Am J Respir Crit Care Med
Pages: 1901-1914 
Number: 10
Work Type: Original
Location: Assoziierter Partner, BREATH, CPC-M
Disease Area: CFBE
Partner / Member: BIH, KUM, MHH
Access-Number: 40209082


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