Pharmaxis Ltd (ASX:PXS) has received a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia for expanded reimbursement of its drug Bronchitol® for the treatment of cystic fibrosis (CF).
Bronchitol® has the active ingredient mannitol delivered as an inhalable dry powder, which restores airway surface liquid.
The PBAC, the government’s independent advisory body on medicine reimbursement, has cleared the way for Pharmaceutical Benefits Scheme (PBS) funding of Bronchitol in combination with reimbursed Pulmozyme® (another CF medication) after considering a submission from Pharmaxis at its most recent meeting.
Gary Phillips, chief executive officer for Pharmaxis, commented:
"The submission for broader access to Bronchitol was strongly supported by the cystic fibrosis community with patients, families, patient organisations and CF clinic teams taking part in the feedback process.
"We look forward to discussing the positive PBAC recommendation with the Australian government to enable the PBS listing that will allow appropriate patients taking Pulmozyme to add reimbursed Bronchitol to their treatment regime."
Nettie Burke, chief executive officer for Cystic Fibrosis Australia (CFA), added:
"Every individual with CF should have access to a variety of treatments which are tailored to their disease and situation.
"The Bronchitol delivery device is portable, and CFA strongly advocates for better, less burdensome treatments that empower people with CF to lead productive lives.
"We are pleased that the PBAC has made this recommendation and anticipate an early listing on the PBS."
A resubmission to the FDA is expected in 2018 seeking approval in the US.
Importance of Bronchitol®
Bronchitol® is an inhaled dry powder delivered via a small handheld inhaler.
It has been studied in more than 1000 patients with CF in clinical trials conducted around the world.
People with CF develop an abnormal amount of excessively thick and sticky mucus within the lungs, airways and the digestive system.
The mucus causes impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections which lead to irreversible damage.
Lung failure is the major cause of death for someone with CF.
The recommendation by the PBAC follows successful registration and reimbursement outcomes in a number of other countries.
Financial strength of Pharmaxis
Pharmaxis is well-funded with $21.5 million in cash at the end of June 2017.
The Boehringer NASH phase 2 initiation milestone is expected in in Q3 2017 for €18 million, and adds to this the Boehringer 2nd indication phase 2 milestone for €10 million expected in H2 2017.
The Pharmaxis share register comprises 50% institutional investors.