Pharmaxis-supported study shows potential for inhaled powder CF treatment

Adding a new long-term inhaled dry powder version of mannitol to standard therapy helps improve lung function in cystic fibrosis patients for up to a year, according to a new study.

But the forced expiratory volume per second between the control groups did not reach clinical significance. Details are published online in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine.

Such a finding is significant because using inhaled mannitol helped improve lung function for up to 12 months and appeared to be safe. The treatment could be used daily, self-administered and was easy to use, according to lead author Moira Aitken, a professor of pulmonary and critical care medicine at the University of Washington Medical Center.

Pharmaxis supported the double-blind study, involving 318 patients receiving 400 mg bid inhaled mannitol, or a control group of 50 mg bid inhaled mannitol for 26 weeks, with 26 additional weeks of open-label active treatments to follow. Scientists also gave the mannitol on top of therapies such as recombinant human deoxyribonuclease or inhaled antibiotics.

Compliance was good in both groups, according to the researchers. The tests showed an 8.22% improvement in forced expiratory volume per second (FEV1) compared with the baseline, versus a 4.47% improvement in the control group. The difference in absolute FEV1 between the study and control groups approached statistical significance, while the difference in relative change from baseline FEV1 between groups reached significance, according to the study. But researchers believe that the difference in absolute forced expiratory volume per second did not reach significance perhaps because of the use of a single baseline visit to establish those values. When baseline FEV1 values were calculated as an average of FEV1 values over two baseline visits, the overall increase in absolute FEV1 was significantly greater in the treatment group, the researchers reported.

The company won Australian regulatory approval this fall for the cystic fibrosis drug bronchitol. FDA approval is pending for the treatment, which is delivered with a hand-held inhaler in dry powder in order to hydrate the lungs, help restore lung clearance and make sure patients can clear their mucus better.

- here's the release
- access the online journal article here

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