FDA Grants Bayer HealthCare Pharmaceuticals Orphan Drug Designation for Investigational Ciprofloxacin Dry Powder Inhaler for the Treatment of Cystic Fibrosis

WAYNE, N.J., March 11 /PRNewswire/ -- Bayer HealthCare Pharmaceuticals announced today that an orphan drug designation has been granted by the U.S. Food and Drug Administration (FDA) for ciprofloxacin dry powder inhaler (DPI) for management of pulmonary infection due to Pseudomonas aeruginosa in cystic fibrosis (CF) patients. A similar designation has already been granted by the European Medicines Agency. Ciprofloxacin DPI is an investigational drug–device combination that combines ciprofloxacin dry powder formulated using Novartis' Proprietary PulmoSphere® technology with a delivery inhaler. Ciprofloxacin DPI is 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.

"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."

Cystic fibrosis is a life-threatening inherited disease affecting the lungs, pancreas, liver, and intestines(1). Approximately 30,000 patients in the US are affected by CF(1). In 2008, the median age of survival for patients in the US was 37.4 years according to data compiled by the Cystic Fibrosis Foundation(2). The major consequences of the disease are pancreatic insufficiency and reduced lung function. Lung disease accounts for about 90 percent of the mortality associated with CF(3). 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(2).

Pulmonary infections in CF patients are a chronic problem and represent the leading cause of exacerbations and mortality(3,4). P. aeruginosa is the leading pathogen in CF patients(5). The thick mucus in the lungs is ideally suited to bacteria, and individuals with CF are colonized and infected by bacteria from an early age; about 20 percent of children under 1 year of age and 80 percent of adult patients with CF have P. aeruginosa present in their sputum(6). Chronic infection with P. aeruginosa is associated with an accelerated decline in pulmonary function, more frequent exacerbations, and increased mortality in patients with CF(3, 7, 8).

Clinical Trials with Ciprofloxacin DPI

In Phase I studies with ciprofloxacin DPI, including those in pediatric(9) 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 (10, 11, 12).  Adverse events reported in the Phase I studies included transient bitter taste after inhalation, a report of transient reduction of FEV1 that resolved without intervention, and one report of bronchospasm related to study drug(13).  A multinational Phase II study evaluating safety and efficacy in CF patients is ongoing with the primary end point of improvement in lung function, measured by FEV1.  An additional clinical study of ciprofloxacin DPI not related to the orphan drug designation for CF is ongoing.  This study in patients with non-CF bronchiectasis is evaluating the safety and efficacy of ciprofloxacin DPI with respect to overall bacterial load and clinical outcomes.  

About Orphan Drug Designation in the United States

Under legislation passed by the U.S. Congress in 1983, the Orphan Drugs Act provides incentives to sponsors to develop products for rare diseases.  The first sponsor who obtains marketing approval for a designated orphan drug or biological product is granted US market exclusivity for a seven-year period.  Exclusivity begins on the date that the marketing application is approved by the FDA for the designated orphan drug and applies only to the indication for which the drug has been designated and approved.  The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and efficacy of a compound must be established through adequate and well-controlled studies.

About Bayer HealthCare Pharmaceuticals

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals unit of Bayer HealthCare LLC, a division of Bayer AG. One of the world's leading, innovative companies in the healthcare and medical products industry, Bayer HealthCare combines the global activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions. In the U.S., Bayer HealthCare Pharmaceuticals comprises the following business units: Women's Healthcare, Diagnostic Imaging, General Medicine, Hematology/Neurology and Oncology. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References

(1)  Cystic Fibrosis Foundation: About cystic fibrosis. Accessible at: http://www.cff.org/AboutCF/, last accessed February 1, 2010

(2)  Cystic Fibrosis Foundation: Frequently asked questions. Accessible at: http://www.cff.org/AboutCF/Faqs/, last accessed February 1, 2010

(3)  Courtney JM, et al. (2007): Predictors of mortality in adults with cystic fibrosis. Pediatr Pulmonol, 42, 525-532

(4)  Amadori A, et al. (2009): Recurrent exacerbations affect FEV1 decline in adult patients with cystic fibrosis. Respir Med, 103, 407-413

(5)  Van Daele SG, et al. (2005): Epidemiology of Pseudomonas aeruginosa in a cystic fibrosis rehabilitation centre. Eur Respir J, 25, 474-481

(6)  Cystic Fibrosis Foundation: Cystic Fibrosis Foundation Patient Registry, 2008 Annual Data Report. Cystic Fibrosis Foundation. Bethesda, MD.

(7)  Kosorok MR, et al. (2001): Acceleration of lung disease in children with cystic fibrosis after Pseudomonas aeruginosa acquisition. Pediatr Pulmonol, 32, 277-287

(8)  Emerson J, et al. (2002): Pseudomonas aeruginosa and other predictors of mortality and morbidity in young children with cystic fibrosis. Pediatr Pulmonol, 34, 91-100

(9)  Data on file – MRR Study 1270

(10)  Stass H, et al. (2008): Pharmacokinetics of inhaled ciprofloxacin powder. Poster presented at the 31st European Cystic Fibrosis Conference, Prague, Czech Republic, 11-14 June 2008

(11)  Stass H, et al. (2008): Ciprofloxacin PulmoSphere® inhalation powder: a healthy volunteer study. Poster presented at the American Thoracic Society International Conference, Toronto, Canada, 16-21 May 2008

(12)  Stass H, et al. (2008): Safety and pharmacokinetics of inhaled dry powder ciprofloxacin after single and multiple inhalations in adult patients with cystic fibrosis. Poster presented at the North American Cystic Fibrosis Conference, Orlando, Florida, 23-25 October 2008

(13)  Data on file – MRR Report No.: PH-36102

SOURCE Bayer HealthCare Pharmaceuticals