Zambon Receives U.S. FDA Breakthrough Therapy Designation for CMS I-neb® in Patients with Non-Cystic Fibrosis Bronchiectasis (NCFB)
- CMS I-neb® is an investigational treatment being developed as a potential first-in-class inhaled therapy for adult patients with NCFB colonized with P. aeruginosa; NCFB is a chronic, progressive, and irreversible respiratory disease
- Breakthrough designation validates registrational path for CMS I-neb® in NCFB and facilitates potentially expedited development and review
MILAN, Italy and Boston, MA, April 21, 2022 — Zambon, a multinational pharmaceutical company focused on innovating cure and care to improve people’s health and the quality of patients’ lives, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to 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 (NCFB) colonized with P. aeruginosa. NCFB 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.
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 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 (QoL). The treatment was demonstrated to be well tolerated with adverse events similar between groups. Data from the Phase 3 trial were most recently presented at the European Respiratory Society (ERS) International Congress in September 2021.
“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. “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.”
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.i
“This FDA Breakthrough Therapy Designation reinforces our confidence in CMS I-neb®, which, if approved, would be the first inhaled treatment for people with NCFB 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.
In patients with NCFB, lung infection with P. aeruginosa is associated with frequent pulmonary exacerbations and admission to hospital for treatment, reduced quality of life, and increased mortality.