Inhaled therapy receives FDA breakthrough designation for non-cystic fibrosis bronchiectasis

The FDA granted breakthrough therapy designation to colistimethate sodium powder for nebulization solution to reduce pulmonary exacerbations in adults with non-cystic fibrosis bronchiectasis and Pseudomonas aeruginosa.

Colistimethate sodium powder for nebulization solution (CMS I-neb, Zambon) is being developed as a potential first-in-class inhaled therapy for adults with non-cystic fibrosis bronchiectasis colonized with P. aeruginosa. CMS I-neb delivers targeted drug concentrations directly to the disease site to minimize systemic exposure that can cause toxicity and adverse events, according to a company press release.

The FDA breakthrough therapy designation was supported by data from the phase 3 PROMIS-I study, which demonstrated CMS I-neb significantly reduced annual pulmonary exacerbation rates compared with placebo in this population. Therapy was also shown to prolong time to first exacerbation and improved quality of life. Treatment was well tolerated and adverse events were similar between groups, according to the release.

Data from the PROMIS-I study were presented at the 2021 European Respiratory Society International Congress.

“With no approved drugs for patients with non-cystic fibrosis 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,” Roberto Tascione, CEO of Zambon, said in the release.

Breakthrough therapy designation is granted to accelerate development and regulatory review of investigational drugs that are intended to treat serious or life-threatening diseases and conditions.

“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,” Paola Castellani, chief medical officer and head of research and development of Zambon, said in the release.