Pharmaxis Concludes Special Protocol Assessment with FDA for Bronchitol Phase 3 Trial
Thursday June 19, 7:05 pm ET
SYDNEY, Australia, June 19 /Xinhua-PRNewswire-FirstCall/ -- Pharmaxis (ASX: PXS - News; Nasdaq: PXSL - News) today announced that the company and the U.S. Food and Drug Administration (FDA) have reached agreement on the phase 3 registration trial of Bronchitol for bronchiectasis via the Special Protocol Assessment process.
The SPA process allows for FDA evaluation of a clinical trial protocol intended to form the primary basis of an efficacy claim in support of a New Drug Application, and provides an agreement that the study design, including trial size, clinical endpoints and/or data analyses are acceptable to the FDA.
Pharmaxis previously agreed on the trial design with the European regulatory agency (EMEA). This trial will therefore form the basis of a marketing application in both the U.S. and Europe.
The phase 3 trial has been designed in collaboration with internationally renowned experts in the field of bronchiectasis and will be a randomized, placebo controlled, double-blind investigation of Bronchitol twice daily in approximately 350 adults with bronchiectasis. Participants will be treated for 52 weeks and the primary endpoints are reduction in frequency of exacerbations and improvement in quality of life. Secondary endpoints include time to first exacerbation and duration of exacerbation. Additional secondary endpoints are antibiotic use, sputum volume, exercise tolerance and lung function measurements. The trial includes health economic analysis and will be conducted in centers across Europe and the U.S.
This trial is the second Phase III study to be undertaken for Bronchitol in bronchiectasis and follows the completion of a successful shorter trial reported last year.
Pharmaxis CEO, Alan Robertson, said the company was pleased to have concluded its discussions with the FDA and the EMEA.
''We believe this phase 3 trial design will allow us to thoroughly demonstrate the clinical benefits of Bronchitol in a patient population for which mucus build up and clearance is a daily problem. Our bronchiectasis program follows closely behind our work in cystic fibrosis where a phase 3 clinical trial is expected to soon close recruitment.''
Pharmaxis is developing Bronchitol as a treatment to improve mucus clearance in the lungs of patients with cystic fibrosis, bronchiectasis and other acute and chronic pulmonary conditions. The U.S. FDA has granted Bronchitol fast track status and it is designated as an orphan drug in the U.S.
surf's up......crikey
