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FDA Grants Zambon Breakthrough Therapy Designation for Therapy for Non-Cystic Fibrosis Bronchiectasis

May 2, 2022

The U.S. Food and Drug Administration granted Zambon Breakthrough Therapy designation for its colistimethate sodium powder for nebulization solution (CMS I-neb) for the reduction in the incidence of pulmonary exacerbations in adult patients with non-cystic fibrosis bronchiectasis colonized with P. aeruginosa.

Non-cystic fibrosis bronchiectasis (NCFB) is a chronic lung disease characterized by recurrent infection, inflammation, persistent cough, and production of sputum and its prevalence is increasing worldwide. NCFB has a progressive course primarily determined by the rate of exacerbations, many of which are related to P. aeruginosa. Consequently, research efforts directed to treat infection by P. aeruginosa and its associated acute exacerbations remain a clinical priority.

Colistimethate sodium (CMS) is a pro-drug (the form used for inhalation therapy) of the antibiotic colistin. Colistin is a polymyxin antibiotic derived from Bacillus polymyxa var. colistinus. The polymyxin antibiotics are surface active agents and act by binding to and changing the permeability of the bacterial cell membrane, causing bacterial cell death. Colistin is an active agent against aerobic Gram-negative pathogens that can cause life- threatening infections, an example being P. aeruginosa. Colistin remains one of the few active antimicrobial agents against multi drug resistant Gram-negative bacteria and is currently considered one of the last therapeutic options for infections such as carbapenem-resistant P. aeruginosa.

The I-neb is a third-generation nebulizer for Adaptive Aerosol Delivery (AAD). The I-neb is a small, battery powered, lightweight and silent drug delivery device, delivering a precise, reproducible dose of the drug. The AAD technology ensures optimal drug delivery by only delivering medication when the patient inhales, (not continuously as in other nebulizers). This gives the medication the best opportunity to reach deep into the lungs and greatly reduces waste to the environment. AAD delivers the right amount of medication, regardless of breath size or breathing pattern.

The Breakthrough Therapy designation is supported by data from the phase 3 PROMIS, which showed that CMS I-neb significantly reduced the annual rate of exacerbations in patients with NCFB 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 NCFB 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.

Breakthrough Therapy Designation from the U.S. FDA is granted to accelerate the development and regulatory review of investigational drugs that are intended to treat serious or life-threatening ailments. Investigational therapies with this designation have shown preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

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