Sydney-based Pharmaxis has announced cystic fibrosis therapy BRONCHITOL (mannitol) met the primary endpoint in a pivotal study required by the FDA for approval in the US.
The study, which was designed to demonstrate the efficacy and safety of BRONCHITOL, was required after the FDA rejected the therapy in 2013.
"The clinical trial (CF303) was designed after extensive consultation with the US Food and Drug Administration (FDA) in order to gain marketing approval for BRONCHITOL to treat adult CF patients in the United States," said the company in a statement.
"The trial was a 26-week randomized, double-blind parallel group investigation of BRONCHITOL administered twice daily in CF patients aged 18 and over to assess improvements in lung function and other parameters, as well as safety. The trial recruited a total of 423 patients across 126 sites in 21 countries including North and South America, Western and Eastern Europe and Australasia. More than a quarter of patients were from the USA."
According to CEO Gary Phillips, “I am pleased that the study met its primary endpoint and whilst the effect size is reduced relative to previous studies Pharmaxis and its US partner Chiesi believe the results are sufficient to underpin a resubmission of the BRONCHITOL New Drug Application to the FDA which we expect will occur in 2018."
BRONCHITOL is already listed on the PBS for the treatment of cystic fibrosis and available through many countries in Europe.
"Incremental improvements in the standard of care for CF have resulted in longer life expectancy and adult patients now exceed 50% of the CF population in many countries. Adult CF patients who experience deteriorating health or difficulty in complying with existing medications continue to require access to new treatment options and in this trial BRONCHITOL brought benefit to patients on top of their existing treatment regimen and had a good safety profile.
“The study results reinforce the body of clinical trial evidence for BRONCHITOL and I’d like to thank both the global CF community and the Pharmaxis team who have delivered a very high quality study with low rates of patient withdrawal in both treatment arms,” added Mr Phillips.
Pharmaxis partners with Italian-based Chiesi for the commercialisation of BRONCHITOL in the US.
Ken McBean, president and CEO of Chiesi US, said, “The conclusion of this key pivotal trial, conducted as a partnership between Pharmaxis and Chiesi R&D teams, provides a foundation for moving towards FDA approval, recognizing the challenge of the agency scrutiny of such assets. We hope, in turn, it can also leverage this new therapeutic option to the adult CF population in the US. If successful, it will also provide a further step towards the growth of our existing portfolio and our business vision in the valuable US market, where our presence is focused on specialty care opportunities including in the field of respiratory medicine, and where we continue to grow through internal pipeline execution and judicious partnering with biotech and pharma companies.”
Mr Phillips added, “We believe the BRONCHITOL business segment based out of our manufacturing facility in Sydney will transition to profitability over the next 12 to 24 months irrespective of any approval in the US. The cash that this will return will help fund Pharmaxis’ drug discovery activities and the exciting pipeline of assets we have developed.”