FDA grants "orphan drug" status to Bayer's Ciprofloxacin dry powder inhaler

FDA grants "orphan drug" status to Bayer's Ciprofloxacin dry powder inhaler for cystic fibrosis

11 March 2010


Mumbai: Bayer Schering Pharma AG, Germany, today said that the US Food and Drug Administration had granted an orphan drug designation for its ciprofloxacin dry powder inhaler (DPI) used in the management of chronic pulmonary infections due to Pseudomonas aeruginosa in cystic fibrosis (CF) patients.

European Medicines Agency (EMA) has already granted a similar designation to Ciprofloxacin DPI, an innovative drug-device combination.

The DPI combines ciprofloxacin dry powder that is formulated using Novartis' Proprietary PulmoSphere technology with an easy-to-use delivery inhaler.

Ciprofloxacin DPI is currently in Phase II development and is being studied for its safety and potential to improve lung function, as measured by the forced expiratory volume in 1 second (FEV1), in patients with CF.

''Receiving the orphan drug designation by the FDA for ciprofloxacin DPI is positive news for patients with this life-threatening disease,'' said Dr. Jean-Philippe Milon, head of global business unit, general medicine, Bayer Schering Pharma AG. ''With ciprofloxacin DPI, we are investigating a promising and convenient treatment option for CF patients worldwide.''

Cystic fibrosis is a life-threatening inherited disease affecting the lungs, pancreas, liver, and intestines. Approximately 30,000 patients in the USA are affected by CF. In 2008, the median age of survival for patients in the USA was 37.4 years according to data compiled by the Cystic Fibrosis Foundation. The major consequences of the disease are pancreatic insufficiency and reduced lung function. Lung disease accounts for about 90 per cent of the mortality associated with CF. Patients with cystic fibrosis have dehydrated, thickened respiratory secretions that are difficult to clear and provide an attractive environment for bacteria, thus increasing the risk of infection and inflammation.

Pulmonary infections in CF patients are a chronic problem and represent the leading cause of exacerbations and mortality. P. aeruginosa is the leading pathogen in CF patients. The thick mucus in the lungs is ideally suited to bacteria, and individuals with CF are colonised and infected by bacteria from an early age; about 20 per cent of children under 1 year of age and 80 per cent of adult patients with CF have P. aeruginosa present in their sputum. Chronic infection with is associated with an accelerated decline in pulmonary function, more frequent exacerbations, and increased mortality in patients with CF.

Novel therapeutic strategies are needed. Current therapies are not curative and CF patients continue to experience significant exacerbations, morbidity, and mortality while on therapy.
Clinical Trials with Ciprofloxacin DPIAccording to Bayer, in Phase I studies with ciprofloxacin DPI in paediatric and adult CF patients, ciprofloxacin has been shown to reach high concentrations in the lung with very low systemic exposure following single and multiple dose administration.

"Initial Phase I study results showed that ciprofloxacin DPI was well tolerated in CF patients without clinically relevant drug treatment related adverse effects," a statement from the company said.

"A multinational Phase II study in CF patients is ongoing with the primary end point of improvement in lung function, measured by FEV1. Additionally, a Phase II study investigating the impact of ciprofloxacin DPI on overall bacterial load and clinical outcomes in patients with non-CF bronchiectasis is also ongoing," the statement added.