The US FDA recently granted Breakthrough Therapy Designation to a novel inhaled therapy called CMS I-neb, which is designed to treat non-CF bronchiectasis, according to pharmaceutical manufacturer Zambon.
CMS I-neb is a colistimethate sodium powder for nebulization solution for the reduction in the incidence of pulmonary exacerbations in adult patients with non-CF bronchiectasis colonized with P. aeruginosa. Non-CF bronchiectasis is a chronic, progressive, and irreversible respiratory disease. There are no approved inhaled treatments currently available for patients with bronchiectasis and chronic P. aeruginosa colonization.
In patients with non-CF bronchiectasis, lung infection with P. aeruginosa is associated with frequent pulmonary exacerbations and admission to hospital for treatment, reduced quality of life, and increased mortality.
The Breakthrough Therapy Designation is supported by data from the Phase 3 PROMIS – I study, which showed that CMS I-neb significantly reduced the annual rate of exacerbations in patients with non-CF bronchiectasis and P. aeruginosa chronic infection, the primary endpoint of the trial.
In addition, the trial met important secondary endpoints, including reduction of severe exacerbations and prolongation of time to first exacerbation compared to placebo, and also improvement in quality of life. The treatment was demonstrated to be well tolerated with adverse events similar between groups.
“With no approved drugs for patients with non-CF bronchiectasis colonized by P. aeruginosa anywhere in the world, the Breakthrough Therapy Designation by FDA marks an important step forward in support of our mission to develop and provide treatment options for people with rare and severe respiratory diseases,” said Roberto Tascione, CEO at Zambon. “We are proud that the FDA has recognized the importance of CMS I-neb and the urgent need to develop innovative treatments for these patients.”
“This FDA Breakthrough Therapy Designation reinforces our confidence in CMS I-neb®, which, if approved, would be the first inhaled treatment for people with non-CF bronchiectasis and chronic P. aeruginosa infection. CMS I-neb is a unique inhaled investigational therapy designed to deliver targeted concentrations of drug directly to the site of disease while aiming to minimize systemic exposure that can cause toxicity and side effects. We look forward to working with the FDA over the coming months as we advance the development of CMS I-neb with the hope of bringing much needed benefit to patients whose lives are severely impacted by this disease,” commented Paola Castellani, CMO and Head of R&D at Zambon.