Bronchitol approved as add on therapy for cystic fibrosis patients

Bronchitol is the first and only inhaled dry powder used to improve pulmonary function in cystic fibrosis (CF) patients as maintenance therapy. It provides a compact, portable option to CF patients. Bronchitol was developed by Pharmaxis Ltd., and Chiesi is now its exclusive distributor in the United States and 11 other countries. It is mostly found to be indicated in cystic fibrosis (CF) patients 18 years of age and older. The use of BRONCHITOL is advised only in adults who have passed the BRONCHITOL Tolerance Test.

Data was collected and analyzed from 3 randomized, double-blind, controlled phase 3 trials in patients with CF. The first trial included a sample size of 423 patients aged 18 years and older with a baseline predicted FEV₁ greater than 40% and less than 90%. However, the 2^nd phase trial assembled 295 patients aged 6 years and older with predicted FEV₁ greater than or equal to 30% and less than 90% whereas the third lot studied 305 patients aged 6 years and older with predicted FEV₁ greater than or equal to 40% and less than 90%.

In these three large-scale global clinical trials totaling 761 patients who were randomized to receive Bronchitol 400mg by oral inhalation or control twice daily; each dose of Bronchitol was preceded by the use of an inhaled short-acting bronchodilator. The primary efficacy end point was framed at analyzing the improvement in lung function as determined by the mean change from baseline in pre-dose FEV₁ (mL) over 26 weeks.

The following key findings were deduced-

1. Bronchitol use led to a sustained improvement in FEV₁ (Forced Expiratory Volume) versus control in the first trial.
2. The statistically significant improvement in FEV₁ was observed over the 26-week treatment period in those patients receiving Bronchitol when compared to patients in the control group.
3. In Trials 2 and 3, the adjusted mean differences in FEV
   1 between Bronchitol and control were 68mL and 52mL, respectively, in the intention-to-treat population.
4. Also, the posthoc descriptive analyses of the adult subgroups in Trials 2 (n=209) and 3 (n=157) showed adjusted mean differences in FEV₁ of 78mL and 78mL, respectively.

The most common adverse reactions observed in 3% or more patients, included cough, hemoptysis, oropharyngeal pain, vomiting, bacteria sputum identified, pyrexia, and arthralgia.

Chiesi USA CEO Ken McBean commented, "Our teams have worked tirelessly toward today's FDA approval, and we are thankful that we stayed the course to arrive at this moment. We're thrilled to achieve this important milestone for the US market and congratulate all Chiesi team members and partners who supported the clinical process."

"Bronchitol offers a portable and discreet option for CF management, with no routine cleaning or maintenance of the inhaler device required," said Dr. Carmen Dell'Anna, Head, Global Medical Excellence at Chiesi. "We are excited to achieve its approval in the U.S. for adults living with cystic fibrosis," she further added.

As a result, Chiesi now says it expects the US launch of Bronchitol to take place in March 2021.