What is the role of ivacaftor (Kalydeco) in the treatment of cystic fibrosis (CF)?

Updated: Oct 22, 2019
  • Author: Girish D Sharma, MD, FCCP, FAAP; Chief Editor: Kenan Haver, MD  more...
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The cystic fibrosis transmembrane conductance regulator (CFTR), ivacaftor (Kalydeco), was approved by the FDA in January 2012. A study by Ramsey et al observed lung function improvement at 2 weeks that was sustained through 48 weeks. The study also observed improvements in risk of pulmonary exacerbations, patient-reported respiratory symptoms, weight gain, and concentration of sweat chloride. [2] Ivacaftor was initially approved for adults and children aged 6 years or older who have at least 1 copy of the G551D mutation in the CFTR gene. In February 2014, ivacaftor gained approval for an additional 8 CFTR gene mutations. [73] In 2015, use in CF was expanded to include children as young as 2 years with the approval of an oral granule which is mixed with soft food or liquid. In 2017, the FDA approved use in children as young as 12 months old. Since its approval, ivacaftor has gradually gained approval for additional CFTR gene mutations, which total 33 as of August 2017.

Approval of lumacaftor/ivacaftor was based on data from 2 Phase III studies (TRAFFIC and TRANSPORT) that enrolled more than 1100 people with CF aged 12 years and older who had 2 copies of the F508del mutation. In September 2016, the FDA expanded this indication to include children aged 6-11 years. People with 2 copies of the F508del mutation represent the largest group of people with CF. Of the 30,000 people in the United States with CF, approximately 8,500 individuals aged 12 years or older have 2 copies of the F508del mutation. Patients treated with lumacaftor/ivacaftor experienced statistically significant improvements in lung function. Patients also experienced reductions in pulmonary exacerbations and improvements in body mass index (BMI). [63]

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