Pharmaxis cystic fibrosis drug given orphan status in Europe

Orphan designation has been granted on the basis of Bronchitol’s potential to treat cystic fibrosis, a genetic disease which, for the patient, is characterized by recurring respiratory complications. Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration.

“This European Orphan designation is another major step forward in the development of Bronchitol,” commented Alan Robertson, Pharmaxis CEO. “The assistance from the EMEA in the final development of Bronchitol will be invaluable in bringing this product to the cystic fibrosis community.”

A recent phase II trial of the drug in cystic fibrosis patients demonstrated statistically significant improvements in lung function for patients being treated with Bronchitol. Pharmaxis is conducting an additional phase II trial aimed at determining the optimal Bronchitol dose for cystic fibrosis and is planning to commence the final international phase III clinical trials in 2006.