FDA rejects Pharmaxis’s NDA for Bronchitol

By Dylan Bushell-Embling
Wednesday, 20 March, 2013

Shares in Pharmaxis (ASX:PXS) fell 7% yesterday after it revealed the US FDA has rejected its New Drug Application for Bronchitol in its current state.

The agency has issued Pharmaxis with a Complete Response Letter advising that Bronchitol cannot yet be approved as a cystic fibrosis treatment for patients six years or older.

The FDA recommended that Pharmaxis conduct another clinical trial, which could significantly prolong the application process.

Pharmaxis said it has been advised that the efficacy data presented from the first two phase III clinical trials are not adequate. For the first trial, this was because of the frequency of treatment-related early dropouts, while for the second one it was due to the lack of statistical significance from the analysis.

The FDA has also raised safety concerns related to the occurrence of haemoptysis, or coughing up of blood, particularly in paediatric patients.

The agency’s decision is consistent with the negative recommendation from an FDA advisory committee reviewing the application in January.

Pharmaxis CEO Gary Phillips said the company is “clearly disappointed that Bronchitol cannot yet be made available to patients in the US”.

He said the company plans a meeting with the FDA next quarter to discuss the parameters of an additional clinical trial.

“The company remains committed to bringing Bronchitol to cystic fibrosis 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.”

Bronchitol is a proprietary formulation of mannitol delivered to the lungs as a dry powder. It is designed to hydrate the lungs and aid with mucus clearance.

Bronchitol has marketing approval as a cystic fibrosis treatment in Australia for patients six years and older, and in the EU for patients 18 years and up.

Pharmaxis shares fell 7.07% on Tuesday to $0.460 after the knockback was announced. The price showed signs of recovering by around 1 pm on Wednesday, trading 2.17% higher at $0.470.