Cystic Fibrosis - Orphan Drug Designation for Innovative Treatment Against Lung Infections

An innovative treatment for infections of the respiratory tract in cystic fibrosis patients has received orphan drug designation in the US.

Axentis Pharma of Zurich, Switzerland announced today that this sought-after designation has been granted to its product candidate Fluidosomes-tobramycin, a therapeutic that will soon be tested in Phase II clinical trials. The company has now been granted orphan drug designation for this candidate in both Europe and the US.

Axentis Pharma (Switzerland) announced today that the Office of Orphan Products Development of the US Food and Drug Administration (FDA) has granted the orphan drug designation to its lead product candidate Fluidosomes-tobramycin. This drug is a liposomal formulation of tobramycin, an innovative treatment for infections of the respiratory tract in patients with cystic fibrosis that is delivered directly to the site of infection via standard nebulizers. Pre-clinical and Phase I clinical studies support improved safety and efficacy profiles for Fluidosomes-tobramycin as compared to currently marketed treatments for infections of the respiratory tract in patients with cystic fibrosis.

The orphan drug designation is granted with respect to treatment of pulmonary infections caused by Pseudomonas aeruginosa, a bacterium that is one of the most common causes of infections of the respiratory tract in patients with cystic fibrosis. Axentis Pharmas product candidate received the orphan drug designation for the US only two months after the application and less than one year after orphan designation in Europe was transferred to the company. Dr. Helmut Brunar, company CEO and President, comments: "The orphan drug designation for the US is very good news for affected patients as well as for Axentis Pharma's shareholders. Together with the Orphan Drug Designation that was already achieved last year in Europe, the US .

Source : www.bio-medicine.org