According to Pharmaxis, the FDA has issued a complete response letter regarding its Bronchitol inhaled mannitol for the treatment of cystic fibrosis. The company says that the FDA requested another clinical trial.
The Pharmaxis press release quotes the CRL, which says, “The submitted data do not provide a favorable benefit‐risk balance to support the use of inhaled mannitol in patients with cystic fibrosis 6 years of age and older. The determination of efficacy based on the two clinical trials are not adequate because of the treatment‐related frequent early dropouts in trial 301 for which the primary statistical analyses did not account and the lack of statistical significance in trial 302 for the primary endpoint.“ The FDA also expressed concern about patients coughing up blood, particularly pediatric patients.
Pharmaxis CEO Gary Phillips commented, “We are clearly disappointed that Bronchitol cannot yet be made available to patients in the US. The FDA has provided guidance on the necessary measures to gain approval and Pharmaxis will now have a follow up meeting with the FDA. This will be a Type A meeting which I expect will take place next quarter and will examine the parameters of an additional clinical trial including how best to incorporate both adult and pediatric patients.
He continued, “At the recent Pulmonary‐Allergy Drugs Advisory Committee (PADAC) meeting we received strong support from the US CF Foundation, leading CF clinicians and patients who spoke passionately about the need for Bronchitol. The Company remains committed to bringing Bronchitol to CF patients in the United States and the onus is now on Pharmaxis to work with the FDA to ensure Bronchitol is approved as soon as possible.”
PADAC issued a negative recommendation on Bronchitol in January 2013.
Read the Pharmaxis press release.
