囊肿性纤维化病属于遗传性疾病，患者体内会产生比正常人更浓稠的粘液，阻碍肺部和其他器官的功能的正常发挥。
在上述实验中，受试者超过300人，每天用药2次共400毫克剂量的Bronchitol进行治疗，为期6个月。在用药6周之后，受试者的肺部功能即得到增强，并且疗效稳定，可以保持到26周。
此外，该药在实验中还达到了次要临床终点，即那些曾采用过或正在使用Pulmozym（Genentech公司产品）的患者使用这种药后，在增强肺功能方面也达到了预定效果。
在欧洲和美国，Bronchitol都已被指定为罕见病治疗药。Pharmaxis计划今年后期递交该药的上市申请。10年来，在全球范围内还没有任何一种治疗囊肿性纤维化病的新药获得批准。
Bronchitol
Overview
Pharmaxis’ key product in development, Bronchitol, is a drug designed to reduce the amount of mucus build-up in the lungs of patients suffering from chronic respiratory conditions.
Pharmaxis is developing Bronchitol for diseases including cystic fibrosis, bronchiectasis and chronic bronchitis. Bronchitol is a proprietary formulation of mannitol administered as a dry powder in a convenient hand-held inhaler. Bronchitol hydrates the lungs, helps restore normal lung clearance, and allows patients to clear mucus more effectively. Clinical studies have shown Bronchitol to be effective and well tolerated, to improve quality of life and mucus flow in people with cystic fibrosis and bronchiectasis.
Phase III clinical trials are underway for the use of Bronchitol in cystic fibrosis. Bronchitol improves the lung function of people with cystic fibrosis, and the clinical trials are designed to show that Bronchitol helps with lung hygiene and improves quality of life for sufferers, who number 75,000 worldwide.
There have been no therapeutic advances to help clear congested lungs for patients with cystic fibrosis for over a decade, and based on positive Phase II trial results, the product has been awarded fast-track status in the US, and orphan drug designation in both the US and EU.
The product is also being developed for use in bronchiectasis, with a pivotal Phase III clinical trial finding that it delivered a highly significant improvement in users’ quality of life.
Bronchiectasis is a progressive lung disease affecting 580,000 people worldwide, and Bronchitol is the first targeted medication for sufferers in over 20 years. Bronchiectasis causes significant disruption to sufferers’ day-to-day lives. Mucus accumulation affects their ability to breathe, exercise, sleep and lead a normal life — ultimately reducing life expectancy.
The drug is supplied on a compassionate use basis to patients throughout Australia. Bronchitol has orphan drug status in the U.S.. The medicine is expected to be approved for bronchiectasis first in Australia, and later in the U.S. and Europe following the completion of a further Phase III trial.
2008/9 Outlook for Bronchitol
Cystic fibrosis
- Complete first global Phase III clinical trial (completed enrolment August 2008)
- Commenced second global Phase III trial in September 2008
Bronchiectasis
- Commence US and European Phase III trial
- Filed marketing application with Australian TGA in September 2008