FDA Approves Orphan Drug Status for Inhaled Ciprofloxacin for Cystic Fibrosis

Yael Waknine

March 16, 2010

March 16, 2010 — The US Food and Drug Administration (FDA) has approved orphan drug status for inhaled ciprofloxacin (Bayer HealthCare Pharmaceuticals) for pulmonary infection resulting from Pseudomonas aeruginosa infection in patients with cystic fibrosis (CF).

The product is formulated using proprietary PulmoSphere technology and administered using a small, portable dry powder inhaler (DPI).

P aeruginosa colonization occurs in about 90% of patients with CF at some point during their lives and is an important contributor to the vicious cycle of infection and inflammation leading to bronchiectasis and eventual respiratory failure. Ciprofloxacin is a fluoroquinolone antibiotic with proven antipseudomonal activity that can decrease the incidence of airway infection, thereby contributing to improvements in long-term prognosis.

In phase 1 studies of pediatric and adult patients with CF, single and multiple doses of ciprofloxacin DPI yielded high pulmonary concentrations with very low systemic exposure.

The most commonly reported adverse events included transient bitter taste after inhalation, a case of transient decrease in forced expiratory volume in 1 minute that resolved without intervention, and a report of drug-related bronchospasm.

According to a company news release, the product is currently in phase 2 multinational trials to evaluate its efficacy for improving CF lung function as measured by forced expiratory volume in 1 minute.

"There continue to be significant unmet medical needs for people with cystic fibrosis," said Shannon Campbell, vice president and general manager, Oncology and General Medicine, Bayer HealthCare Pharmaceuticals. "We are pleased to receive the orphan drug designation from the FDA for ciprofloxacin DPI, which we are researching as a potential treatment option for management of pulmonary infections due to P. aeruginosa in CF patients."

A similar designation for ciprofloxacin DPI previously was approved in the European Union.


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