Pharmaxis (ASX: PXS) is a specialty pharmaceutical company focused on the development of new products for the diagnosis and treatment of chronic respiratory and immune disorders.
Pharmaxis (ASX: PXS) is expecting to receive European marketing authorisation for its cystic fibrosis drug in January next year after the Committee for Medicinal Products for Human Use (CHMP) recommended it be granted.
Pharmaxis expects the European Commission to support the decision and grant the marketing authorisation for Bronchitol for the treatment of cystic fibrosis in patients aged 18 and above.
Bronchitol increases mucociliary clearance – the self-clearing mechanism of the bronchi – and will be the first approved therapy in Europe capable of doing this. It has been shown to improve both lung function and reduce exacerbations when combined with standard medications in use today.
CHMP initially refused to recommend marketing authorisation be granted for Bronchitol in June because it did not believe the effectiveness and benefit of the drug had been established.
Pharmaxis subsequently requested a re‐examination of the European Bronchitol marketing application in early July.
Bronchitol will be used for cystic fibrosis patients aged 18 and above, which represents about two thirds of all patients who could potentially benefit from the drug.
Pharmaxis will undertake a short term clinical trial in children, aged 6‐17, with a view to extending the licence to this age group.
Bronchitol is a drug which was discovered and developed in Australia. It is approved for marketing in Australia and is now set to become available throughout the 27 countries of the European Union.
Pharmaxis will move quickly to commercialise Bronchitol in Europe. The company has established its supply and logistics arrangements and advanced launch preparations and pricing applications with marketing partner, Quintiles.
The European Cystic Fibrosis Society and the American Cystic Fibrosis Foundation have both welcomed CHMP’s decision to grant marketing authorisation.
European Cystic Fibrosis Society president Professor Stuart Elborn said, “Life expectancy in cystic fibrosis is improving but there remains an urgent need for new therapies which can improve lung function and reduce exacerbations.”
Meanwhile, American Cystic Fibrosis Foundation president and chief executive officer Robert J. Beall said the organisation looks forward to Bronchitol being submitted to the U.S. Food and Drug Administration early next year.
Bronchitol’s approval for the treatment of cystic fibrosis patients in Australia, and the European marketing application are based on the results of two Phase 3 clinical trials involving more than 600 people, conducted in 95 centres globally.