Pharmaxis (ASX: PXS) has received a positive recommendation from the National Institute for Health and Clinical Excellence in the UK for its cystic fibrosis treatment, Bronchitol®, which clears the way for reimbursement by the National Health Service.
Bronchitol®, which is licensed in the UK for the treatment of adult cystic fibrosis patients, is the first and only cystic fibrosis product to have received a positive recommendation from the National Institute for Health and Clinical Excellence.
The institute provides evidence‐based guidance to help resolve uncertainty about which medicines represent the best quality care and the best value for money for the UK’s National Health Service.
Cystic fibrosis is a genetic disorder affecting around 40,000 people in Western Europe, 30,000 in the U.S. and 3,000 in Australia.
Bronchitol® has Orphan Drug Designation in the U.S., Europe and Australia and is approved for marketing in Australia and throughout the European Union.
An application has been submitted for marketing approval in the U.S., with a review by the U.S. Food and Drug Administration scheduled for completion in March 2013.
The recommendation provides a new option for cystic fibrosis patients who cannot use recombinant human deoxyribonuclease because of ineligibility, intolerance or inadequate response, a decline in lung function and where other osmotic agents are not considered appropriate.
This new treatment has the potential to delay lung function decline in cystic fibrosis patients, thereby reducing the associated risks of exacerbation and mortality.
Bronchitol® function
Bronchitol® has been developed to help clear mucus, a major source of lung infections, improve lung function and reduce exacerbations in patients with cystic fibrosis.
The treatment is a proprietary formulation of mannitol administered as a dry powder in a handheld inhaler.
It hydrates the lungs, helps restore normal lung clearance, and allows patients to clear mucus more effectively.
Clinical studies have shown Bronchitol® to be effective and well tolerated in treating patients with cystic fibrosis.
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