Pharmaxis has announced that its pivotal Phase 3 trial of Bronchitol mannitol DPI in adult patients with cystic fibrosis met its primary endpoint of change in FEV1 from baseline over the 26-week treatment period; however the company said that the improvement was less than that reported in previous Phase 3 studies in adults.
Pharmaxis, which is partnered with Chiesi for Bronchitol in the US, announced completion of the trial in February 2017.
The FDA issued a CRL for the company’s NDA for Bronchitol in 2013, saying that there were insufficient data to support its use in CF patients aged 6 and older, and this study was designed to satisfy the FDA’s request for additional data. Pharmaxis now says Chiesi USA will submit an updated NDA for Bronchitol next year.
Pharmaxis CEO Gary Phillips commented, “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. 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.”
Chiesi USA President and CEO Ken McBean 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.”
Read the Pharmaxis press release.
