Vertex Pharmaceuticals Inc presented new data on Kalydeco (ivacaftor) data at the 37th European Cystic Fibrosis Society (ECFS) Conference from June 11-14 in Gothenburg, Sweden.
New data from an analysis of Phase 3 and long-term extension studies in people with cystic fibrosis (CF) who have the G551D mutation showed that treatment with the drug reduced the annual loss of lung function by half over three years, compared to similar untreated CF patients.
In addition, the first data from a rollover study following the Phase 3 KONDUCT study in people with the R117H mutation confirmed earlier results that demonstrated lung function improvements in people ages 18 and older. New data were also presented for the first time from the second part of the Phase 3 KONNECTION study in people with certain non-G551D gating mutations and showed that previously reported lung function improvements were maintained through 24 weeks of treatment.
“People with cystic fibrosis experience significant and progressive loss of lung function during their lives. This decline in lung function is the primary cause of death in people with CF, and more rapid loss of lung function is associated with earlier death,” said Jeffrey Chodakewitz, MD, senior vice president and chief medical officer at Vertex.
“Not only does KALYDECO lead to significant initial improvements, but these new long-term data showed that it also appeared to modify the rate of lung function decline, reducing the loss of lung function in people with the G551D mutation. These findings, together with new data from studies in people with the R117H mutation and certain non-G551D gating mutations, show our continued progress in evaluating the benefits of this medicine across different groups of patients and also show that our approach to treating the underlying cause of CF can provide benefits for people with this rare and devastating disease.”
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