By Datamonitor staff writer
Fluidosomes-tobramycin, a liposomal formulation of tobramycin
Axentis Pharma has received the orphan drug designation for its lead product candidate Fluidosomes-tobramycin from the Office of Orphan Products Development of the FDA.
According to Axentis, this drug is a liposomal formulation of tobramycin, a treatment for infections of the respiratory tract in patients with cystic fibrosis that is delivered directly to the site of infection via standard nebulizers.
Preclinical 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 company said.
Currently, the company is initiating Phase II clinical trials that will assess the safety and tolerability of a new therapeutic formulation as well as the effects of two different doses of the new drug. Results of the clinical trial are expected in early 2010.
Helmut Brunar, CEO and president of Axentis, said: "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 designation puts Axentis Pharma in a favorable position to register Fluidosomes-tobramycin in two major world markets with substantial support of the relevant authorities and at a cost advantage for the company.
"As a result, we will be able to deliver the product at competitive prices to patients once it has passed the final clinical test phase. In addition to this, the orphan drug designation grants Axentis several years of exclusive marketing rights once the product has been launched. That is a significant strengthening of Axentis Pharma's market position as well as the company's value."