Pharmaxis has reached its targeted enrollment in its international clinical trial (CF303) evaluating Bronchitol (mannitol) in cystic fibrosis (CF) patients.
The Australian pharmaceutical company expects results by the middle of 2017. The Phase 3 clinical study is compliant with U.S. Food and Drug Administration (FDA) requirements, so the company can get approval for Bronchitol as a treatment for CF in adult patients in the United States.
If the trial reports positive outcomes, the company will submit a response letter to the FDA, and a decision could be expected by 2018.
Bronchitol is a dry powder form of mannitol that is delivered to the lungs via portable inhaler. Meant to be taken twice daily, Bronchitol works by rehydrating the airway/lung surface and promoting a productive cough. The company reports that previous trials have shown that Bronchitol helps to increase mucus clearance and improve lung function.
The product is approved in Australia for patients older than 6, and in the European Union and Israel for patients age 18 and older.
The trial, titled “DPM‐CF‐303: Long Term Administration of Inhaled Mannitol in Cystic Fibrosis – A Safety and Efficacy Trial in Adult Cystic Fibrosis Subjects,” is a 26-week randomized, double-blind, parallel group investigation of Bronchitol administered twice-daily in adult CF patients. The study will assess improvements in lung function, pulmonary exacerbations, and drug safety.
After randomizing all patients screened at the trial sites, final enrollment could reach 420 adult cystic fibrosis patients.
Pharmaxis has partnered with Chiesi Farmaceutici SpA for the development of Bronchitol’s approval in the U.S. Chiesi was a major supporter of the trial, paying $22 million of the total expected $26 million. Chiesi will also complete the FDA’s New Drug Application (NDA) submission for Bronchitol. The therapy, if approved by the FDA, will be integrated into Chiesi’s cystic fibrosis portfolio.
“We are pleased to have attained this significant milestone in such a large undertaking, securing more than 400 study participants at 126 sites in 21 countries. The U.S. is the largest CF market and we now have greater certainty around when the study will report and, subject to that report being positive, when the FDA will complete its consideration of our new drug application,” Pharmaxis CEO Gary Phillips said in a press release.
He said the clinical study protocol closely follows the design of the two large-scale clinical trials already undertaken by Pharmaxis (CF 301 and CF 302) where a post-hoc analysis of the subgroups of the 307 adult patients showed a significant improvement in FEV1.
Phillips thanked the CF community for its support and participation. “We look forward to working with Chiesi to make this new treatment option available for CF patients in the United States,” he added.