Study Results

• What We Learned:

  Orkambi® treatment using weight-adjusted doses appeared to be generally safe and well tolerated in children ages 1 to less than 2 years old and resulted in reduced sweat chloride. The safety profile was consistent with that observed in older age groups. Children showed improved biomarkers of pancreatic function and appeared to have normal growth during the treatment period. 

• Primary Findings:

  Effectiveness:

  This study was conducted between September 2018 and October 2021.

  Part A of this study recruited 14 participants, of whom one discontinued treatment due to a rash while all other participants completed the treatment period and confirmed the dose levels were appropriate for Part B. Part B recruited 46 participants of whom one discontinued treatment due to increased liver enzymes while all other participants completed the treatment period. 

   

  Orkambi® treatmen for 24 weeks resulted in a decrease in sweat chloride of -29.1 mmol/L. This decrease was similar in magnitude to what was observed in the older participants of the phase 3 trials of Orkambi®.

  Safety:

  Orkambi® weight-adjusted doses appeared to be generally well-tolerated in children ages 1 to less than 2 years. Elevated liver function enzymes were noted for 11% of participants. The rate of elevated liver function enzymes in this group indicated that frequent monitoring of liver function should occur in young children using this drug, particularly those with a history of elevated liver function enzymes.

• Citation:

  Am J Respir Crit Care Med 2022;206(10):1239-1247

For current information about the overall development status of this drug, please check the Drug Development Pipeline.