Study Results

• What We Learned:

  This study found that participants, who received mannitol, over a 26-week treatment period, had greater improvements in lung function than those who received placebo. Mannitol was well-tolerated and has an adverse event profile similar to that of the placebo group. The drug was approved by the FDA as add-on maintenance therapy to improve pulmonary function in adult patients 18 years and older with cystic fibrosis (CF). — Bronchitol should only be used in adults who have passed the Bronchitol Tolerance Test.

• Primary Findings:

  Effectiveness:

  This study was conducted between September 2014 and February 2017. The study enrolled 423 participants in 21 countries with more than a quarter of the participants in the USA. The study enrolled 423 patients into two groups: inhaled mannitol 400mg twice a day or a control group that received inhaled mannitol 50mg twice a day. Both the active and control mannitol doses were administered by dry powder inhalation.

   

  The primary goal of the study was to evaluate the effect of mannitol on change in lung function (measured by FEV1) from baseline over the 26-week treatment period. The study showed that participants who received mannitol had greater improvement in lung function, over the 26 week period, than the participants that received placebo (2.2% relative change in FEV1, p=0.025).

   

  The primary outcome was the average FEV1 over the 26-week treatment period. Mannitol was statistically significantly better than control over this period with a mean difference of 54mL. Additional analyses were similar, showing a greater percent predicted FEV1 in the mannitol group of 1.21 points.

   

  A key secondary outcome was the need for antibiotics to treat pulmonary exacerbation. This did not differ between the two groups

  Safety:

  The number of all adverse events and treatment-related adverse events were not statistically significantly different between the two groups

• Citation:

  J Cyst Fibros 2021;20(6):1003-1009

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