Mannitol (brand name Bronchitol) is a dry powder for inhalation, being developed by Pharmaxis, Inc.1 It works by drawing water into the airways in people with CF, thus moistening and thinning the sticky mucus that people with CF have, helping them cough easier. The therapy has been approved for the treatment of people with CF over six years old in Australia and over 18 throughout the European Union and in Israel.2

Mannitol clinical studies

Pharmaxis announced in July 2016 that it has completed recruiting participants with CF for a large and potentially pivotal Phase 3 clinical trial (NCT02134353) to provide evidence of the safety and efficacy of mannitol 400mg twice daily in adult participants over 18 years old. The results are expected in the second quarter of 2017 in accordance with the requirements of the FDA in order to obtain approval in the US in adults with CF.

The study is a 26 week, randomized and double-blind parallel group and besides safety and efficacy, its objectives include improvement in lung function and pulmonary exacerbations as measured by changes in forced expiration (FEV1) at week 6, 14 and 26.

The most common side effect people experience is cough (happens in more than 1 patient in 10). The most serious side effect is bronchospasm, which is the narrowing of the airways of the lung during the first treatments and coughing of blood.4

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