Pharmaxis Ltd’s (ASX:PXS) US licensee Chiesi Group has received a complete response letter from the US Food and Drug Administration (FDA) detailing the remaining matters to be addressed before Bronchitol® can be approved for adult cystic fibrosis (CF) patients in the US.
Based upon the clear and achievable path to approval communicated in the FDA complete response letter, Pharmaxis believes that the FDA review of the Bronchitol NDA (New Drug Application) will be completed in Q1 2020.
The main requirement included in the FDA complete response letter is that Chiesi revise the product packaging and user instructions; and then conduct a human factor study (HFS) demonstrating that the revised user components enable healthcare professionals to properly administer the mannitol tolerance test.
Pharmaxis CEO Gary Phillips said: “We are confident that the FDA complete response letter provides a clear and achievable path to approval.
“FDA requested changes to the product packaging and user instructions require that an HFS be conducted. This is targeted for completion by the end of 2019.
“Based on our experience with healthcare professionals in other markets where our training and packaging has supported thousands of mannitol tolerance tests that have been conducted to ensure patients hypersensitive to mannitol are not prescribed Bronchitol, we are very confident the requested FDA changes can be efficiently implemented and will be effective in achieving the desired goal.
“We have been sharing our experiences in other markets with Chiesi and continue to work collaboratively to prepare for a successful introduction to patients in US cystic fibrosis clinics.”
Bronchitol is an inhaled dry powder for the treatment of cystic fibrosis and has been the subject of three large scale global clinical trials conducted by Pharmaxis.
It is approved and marketed in Europe, Russia, Australia and several other countries.
Chiesi is responsible for the regulatory approval and commercialisation of Bronchitol in the US.
If Bronchitol is approved by the FDA, Pharmaxis will receive a US$10 million milestone payment on the commercial launch of Bronchitol in the US and mid to high teen percentage royalties on in‐market net sales.
Pharmaxis will manufacture and be the exclusive supplier of Bronchitol for the US market.