US FAST-TRACKS PHARMAXIS CYSTIC FIBROSIS DRUG.Pharmaceutical company Pharmaxis (NASDAQ:PXSL), Sidney, Australia, has announced that the US Food and Drug Administration has designated Bronchitol as a fast-track product for cystic fibrosis cystic fibrosis (sĭs`tĭk fībrō`sĭs), inherited disorder of the exocrine glands (see gland), affecting children and young people; median survival is 25 years in females and 30 years in males.. The FDA fast-tracks the New Drug Application (NDA) process if a therapy can potentially address an unmet medical need for a life-threatening disease. Designation as a fast-track product is designed to expedite regulatory review of the Bronchitol NDA. The FDA and European Medicines Agency have both previously granted Bronchitol orphan drug status for treating cystic fibrosis. Pharmaxis chief executive officer Dr Alan Robertson said: "The FDA decision is encouraging news for thousands of cystic fibrosis patients. It recognises the positive clinical data for Bronchitol in treating cystic fibrosis, which is a lethal inherited condition. "This designation will speed the process of bringing this potentially life-saving drug to cystic fibrosis sufferers. "Phase II studies show Bronchitol significantly improves lung function and wellbeing in patients with cystic fibrosis. We look forward to working with the FDA on introducing Bronchitol to the U.S. market as rapidly as possible." Pharmaxis is currently conducting Phase III and II clinical trials of Bronchitol in cystic fibrosis patients at sites in Europe, Canada, Argentina and Australia. Cystic fibrosis affects approximately 75,000 people in the developed world, including 33,000 US patients and 2,500 Australians -- a fifth of whom are children under five years old. There have been no treatment advances in over a decade, and no products are approved to improve lung hydration. Designation as a fast track product enables Pharmaxis to file the new drug application on a rolling basis as data becomes available, allowing the FDA to review the application in sections ahead of receiving the complete submission. A complete submission is expected to be made in 2008. Pharmaxis is developing Bronchitol as a treatment to improve mucus clearance in the lungs of patients with cystic fibrosis, bronchiectasis bron  chi·ec·tat ic (- k-t t and chronic obstructive pulmonary diseases. Bronchitol is a patented, inhalable dry powder Dry Powder A slang term for cash reserves kept on hand to cover future obligations.Notes: For example, if a venture capitalist expects bad times in the IPO markets you might hear him say something like, "we want to keep enough dry powder around to keep funding our companies through these hard times." See also: Burn Rate, Seed Capital, Venture Capital formulation of mannitol mannitol /man·ni·tol/ (man´i-tol) a sugar alcohol formed by reduction of mannose or fructose and widely distributed in plants and fungi; an osmotic diuretic used to prevent and treat acute renal failure, to promote excretion of toxic substances, to reduce cerebral edema or elevated intracranial or intraocular pressure, and to prevent hemolysis during transurethral surgical procedures. administered by a hand-held, pocket sized device. For more information, visit http://www.pharmaxis.com.au or call 212/477-9007. |
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