Study Results

• What We Learned:

  Study results show that there was a significant improvement in lung function in both lumacaftor-ivacaftor dose groups compared to placebo. Participants treated with lumacaftor-ivacaftor experienced more shortness of breath, chest tightness and abnormal liver function.

• Primary Findings:

  Effectiveness:

  The results presented are the combined results of the VX-809-103 and 104 trials. These two Phase 3 trials studies were conducted between April 2013 and April 2014 and enrolled 1,108 patients aged 12 years and older with two copies of the F508del CFTR mutation. The majority of patients completed their assigned study regimens: 348 patients in the lumacaftor (600 mg/day)–ivacaftor group (94.6%), 344 patients in the lumacaftor (400 mg every 12 hr)–ivacaftor group (93.2%), and 362 patients in the placebo group (97.6%) The primary endpoint, absolute change in FEV1 from baseline to week 24, was met. Both dose groups showed significant improvement over placebo-treated participants in this measurement (3.3% improvement for the lumacaftor (600 mg/day)–ivacaftor group and 2.8% improvement for the lumacaftor (400 mg every 12 hr)–ivacaftor group).

   

  The rate of pulmonary exacerbations was lower in both lumacaftor-ivacaftor dose groups than in the placebo group; 30% reduction for lumacaftor (600 mg/day)–ivacaftor group and 39% reduction for the lumacaftor (400 mg every 12 hr)–ivacaftor group. Additionally weight increased steadily for both lumacaftor-ivacaftor dose groups over the 24 weeks of the study.

  Safety:

  The incidence of reported adverse events was similar across the lumacaftor-ivacaftor groups and the placebo groups. Events reported more frequently in lumacaftor-ivacaftor treated participants included shortness of breath, chest tightness and abnormal liver function.

• Citation:

  N Eng J Med ;DOI doi: 10.1056/NEJMoa1409547

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