Bronchitol, Inhalation Powder for Mucus Clearance, Arriving in US

Pharmaxis announced that a first batch of Bronchitol (mannitol), its dry powder and mucus clearing treatment for cystic fibrosis (CF), is on its way to the U.S. from the company’s manufacturing center in Australia.

Recently approved by U.S. Food and Drug Administration (FDA) as a maintenance treatment for adults, it is being marketed by Chiesi USA, its exclusive U.S. distributor.

“This represents a proud and very rare achievement for a home-grown pharmaceutical research company,” Gary Phillips, CEO of Pharmaxis, said in a press release.

“Not only did the team at Pharmaxis design, lead and complete the three large scale international clinical trials which established Bronchitol as a safe and effective medication for CF patients, we have now manufactured the drug to be used by adult CF patients in the USA,” Phillips added.

Bronchitol’s active ingredient, mannitol, is a diuretic with the ability to attract water. Administered using a small and portable dry powder inhaler, mannitol draws water into the airways, rehydrating them and thinning the sticky mucus that is typical in CF patients, making it easier to expel through productive cough.

By increasing mucus clearance, this therapy helps to improve lung function and quality of life in people with CF.

“Production of drugs for delivery to the lungs is one of the most difficult processes undertaken in medicine manufacturing,” Phillips said.

His company’s factory, based in a suburb of Sydney, “is equipped with the technology to engineer a powder with precise control of the particle size, suitable for delivery via a hand‐held inhaler. The powder is put into capsules, and then packaged with all the information required for use by CF patients who will take the drug twice a day.”

“I’d like to thank our production staff for their unwavering dedication and we look forward to servicing the US market with a new treatment option in CF,” he added.

Bronchitol was approved in the U.S. as a maintenance treatment for adults, ages 18 and older with clinically stable disease, based on findings from three Phase 3 trials (NCT00630812, NCT00446680, and NCT02134353). Results overall showed patients given Bronchitol had a significant improvement in lung function compared with those in a placebo group.

Before its start as a regular therapy, patients are required to undergo a Bronchitol tolerance test to ensure its use does not cause a bronchospasm (a sudden tightening in the walls of the airways) or otherwise diminish lung capacity and oxygen blood levels.

As part of the agreement, Pharmaxis received an upfront payment of AU$10 million (about $7 million) from Chiesi, and will now receive an additional payment AU$4 million as certain predetermined milestones were achieved.

Bronchitol is also approved in countries that include the European Union, Russia, Israel, and Australia.