Pharmaxis drug earns FDA 'orphan' status for cystic fibrosis

The US Food and Drug Administration has expanded an 'orphan drug' designation to Pharmaxis' (ASX:PXS) drug Bronchitol, for the additional indication of facilitating mucus clearance in patients with cystic fibrosis.

Pharmaxis was initially granted orphan drug status for Bronchitol as a treatment for patients with bronchiectasis in February.

Orphan drug status is granted to treatment aimed at serious or life-threatening diseases. Cystic fibrosis patients have a defective gene which causes the body to produce an abnormally thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections.

"There have been no therapeutic advances to help clear congested lungs for patients with cystic fibrosis in the past 10 years," said Pharmaxis CEO Alan Robertson in a statement.

Under orphan drug status, Pharmaxis has a seven-year period of market exclusivity. Other incentives include assistance from the FDA with clinical trial design and an exemption from FDA user fees.