Investor Centre

Pharmaceutical company Pharmaxis (ASX: PXS) today presented a new analysis of its two Phase 3 clinical studies of Bronchitol® showing positive trends in reducing exacerbations in all age sub groups of cystic fibrosis patients.

The analysis which focuses on adult patients, has been presented at the European Cystic Fibrosis Meeting taking place in Ireland.  Its release coincides with the commercial launch of Bronchitol in the UK and Germany following the approval by the European Medicines Agency of Bronchitol earlier this year for the treatment of cystic fibrosis in adults as an add-on therapy to best standard of care.

The two phase 3 studies contained 341 adult patients and their results were broadly consistent with those from the overall population of 600 patients which included adolescents and children.

• Adult patients who showed an increase in lung function on Bronchitol had 59% fewer exacerbations than patients who showed no improvement (p=0.03).
• Exacerbation incidence was reduced by 29% in the overall population (p=0.039) and there were consistent improvements in all age groups with a 24% reduction in adults (NS).
• Sputum weight in adult patients was significantly increased at both 6 and 14 weeks
• Lung function in adults showed significant improvements over the 6 months of the study and were sustained out to 12 months. 
• For adults, there was no increase in treatment burden after taking Bronchitol for 6 months.

NICE DRAFT RECOMMENDATION ON COST EFFECTIVENESS

Pharmaceutical company Pharmaxis (ASX:PXS) today announced that the National Institute for Health and Clinical Excellence (NICE) in the UK has issued a preliminary opinion that Bronchitol (inhaled dry powder mannitol) could not yet be considered a cost-effective use of NHS resources for the treatment of cystic fibrosis in England and Wales.  This opinion is not NICE’s final guidance on Bronchitol and the recommendations may change following consultation and receipt of stakeholder comment.

UK LAUNCH

Bronchitol has been introduced in the United Kingdom at a retail price of £16.55 per day and notification has been received from the UK Department of Health that this price is acceptable.

PRICE IN GERMANY

Pharmaxis today officially published the retail price of Bronchitol in Germany at €35.65 per day.  Germany's Federal Joint Committee (G-BA) has a modified procedure for orphan drug assessments such that only products with annual sales in Germany of more than €50 million will be subject to an early benefit assessment from the Institute for Quality and Efficiency in Health Care (IQWiG).

Pharmaceutical company Pharmaxis (ASX: PXS) is pleased to announce it has submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) seeking approval for Bronchitol® for the treatment of patients with cystic fibrosis in the United States.  The FDA has previously granted Bronchitol Orphan Drug designation for the treatment of patients with cystic fibrosis.