Pharmaxis drug earns FDA 'orphan' status for cystic fibrosis

By Helen Schuller
Wednesday, 20 July, 2005

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.

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