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Bronchitol Granted Orphan Drug Status in Europe
14 / 11 / 2005
Pharmaxis Ltd (ASX:PXS, NASDAQ:PXSL) announced today that the European Medicines Agency (EMEA) has granted Orphan designation to the company's product BronchitolT, for the treatment of cystic fibrosis.
Orphan designation has been granted on the basis of Bronchitol's potential to treat cystic fibrosis, a genetic disease which, for the patient, is characterised by recurring respiratory complications.
Alan Robertson, Pharmaxis chief executive officer said: "This European Orphan designation is another major step forward in the development of Bronchitol. The assistance from the EMEA in the final development of Bronchitol will be invaluable in bringing this product to the cystic fibrosis community."
Our recent Phase II trial in cystic fibrosis patients was successful, and 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.
Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration. European Orphan designation is granted to those products intended for the diagnosis, prevention and treatment of diseases that affect not more than five in 10,000 people in the European Community, or conditions where no current therapy exists. European Orphan designation entitles Pharmaxis to a range of incentives including a ten-year period of market exclusivity, protocol assistance to optimise drug development in the designated indication, reduction in registration fees and eligibilty for grants and initiatives supporting research and development.
Orphan designation has been granted on the basis of Bronchitol's potential to treat cystic fibrosis, a genetic disease which, for the patient, is characterised by recurring respiratory complications.
Alan Robertson, Pharmaxis chief executive officer said: "This European Orphan designation is another major step forward in the development of Bronchitol. The assistance from the EMEA in the final development of Bronchitol will be invaluable in bringing this product to the cystic fibrosis community."
Our recent Phase II trial in cystic fibrosis patients was successful, and 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.
Approximately 75,000 people in the major pharmaceutical markets are affected with cystic fibrosis and no products have been approved to improve lung hydration. European Orphan designation is granted to those products intended for the diagnosis, prevention and treatment of diseases that affect not more than five in 10,000 people in the European Community, or conditions where no current therapy exists. European Orphan designation entitles Pharmaxis to a range of incentives including a ten-year period of market exclusivity, protocol assistance to optimise drug development in the designated indication, reduction in registration fees and eligibilty for grants and initiatives supporting research and development.