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Jul 30, 2013

Vertex CF Drug Shows Promise for a New Set of Patients in Phase III Trial

  • Vertex Pharmaceuticals has released promising Phase III results for people with cystic fibrosis (CF) and at least one non-G551D gating mutation, showing that ivacaftor monotherapy produced statistically significant improvements in lung function among this patient group.

    Vertex’ ivacaftor (Kayldeco) is approved in the U.S. and EU for CF patients six and older who have at least one copy of the G551D mutation in the CF transmembrane conductance regulator (CFTR) gene. In trial results released this week, the firm shows that ivacaftor monotherapy is also effective in improving lung function among a subset of CF patients who have different genetic signatures. Vertex also said that administration of ivacaftor resulted in improved weight gain and patient-reported quality of life, per responses to the Cystic Fibrosis Questionnaire Revised.

    “Our goal in CF is to help as many people as possible with our medicines, and these data are an important step toward that goal,” Robert Kauffman, M.D., Ph.D., svp and CMO, Vertex, said in a statement. “The data announced today in people with CF who have a gating mutation showed a meaningful improvement in lung function and support our plans to seek approval of ivacaftor for these patients later this year.”

    Indeed, Vertex announced its intentions to file a supplemental new drug application in the U.S. and marketing authorization application variation in Europe related to gating mutations during the second half of this year.


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