BURNHAM, England, October 26, 2012 /PRNewswire/ --

The European headquarters of Australian pharmaceutical company Pharmaxis (ASX: PXS) today announced that the National Institute for Health and Clinical Excellence (NICE) has issued a positive recommendation in their Final Appraisal Determination (FAD) for Bronchitol® (mannitol dry powder for inhalation), licensed for the treatment of adult cystic fibrosis (CF) patients aged 18 years and above as an add-on therapy to best standard of care.

Bronchitol® is the first CF product to have been fully reviewed and received a positive recommendation from NICE. The recommendation provides a new option for CF patients critically requiring new treatments and who have the highest unmet medical need. This includes those patients who cannot use rhDNase (recombinant human deoxyribonuclease) because of ineligibility, intolerance or inadequate response, whose lung function is rapidly declining (forced expiratory volume in 1 second [FEV1] with a decline greater than 2% annually) and for whom other osmotic agents are not considered appropriate.

"The clinical studies of Bronchitol® showed very promising results for what is a complex therapeutic area" said Dr Diana Bilton, Director of the Adult Cystic Fibrosis Unit at the Royal Brompton Hospital, London, and lead investigator for the first of the Bronchitol® Phase III trials. "This new treatment has the potential to delay lung function decline in CF patients, thereby reducing the associated risks of exacerbation and mortality. Bronchitol® brings a clear step change in efficacy and ease of use to a patient population that currently spend large amounts of time on laborious nebulised therapies. These patients will be delighted to have a new inhaled treatment option".

CF is one of the most common life-threatening inherited diseases, with over 9,000 people affected in the UK, including approximately 4500 adults. Patients with CF can experience a rapid decline in lung function, with frequent respiratory infections (exacerbations) that can require hospitalisation. As a consequence of progressive loss in lung function, patients often require lung transplantation and suffer premature death. In the UK, the median age of death for patients with CF is 29 years old^[1].  

Jo Osmond, Director of Clinical Care at the Cystic Fibrosis Trust, said "We are very pleased with this decision from NICE, as we firmly believe that Bronchitol® is an important addition to CF care, which will help to relieve the burden of treatment for adults with this debilitating condition."

Current best standard of care in the UK is complex and based upon patients' individual needs. It can represent a significant patient and carer burden, with daily respiratory physiotherapy, nutritional control, inhaled/oral antibiotics, bronchodilators and inhaled/oral corticosteroids. To facilitate mucus clearance, patients may also receive aerosolised rhDNase (Pulmozyme®, and whilst unlicensed, nebulised hypertonic saline may also be given.  

The latest decision from NICE provides guidance on the use of Bronchitol® for the treatment of selected adults with CF who have the greatest unmet medical needs. The next stage of the process is publication of the full, final guidance which requires compliance from Primary Care Trusts in terms of reimbursement and treatment access within a 90-day deadline from the date of publication. Pharmaxis anticipates that full guidance will be published and available on the NICE website by November 28^th, 2012, subject to any appeals or factual corrections received by NICE. (http://guidance.nice.org.uk/TA/Wave21/9)

"We welcome this decision from NICE, which recognises Bronchitol® as a clinically and cost-effective treatment option for CF patients who have the greatest unmet medical need", said Dr Alan Robertson, CEO of Pharmaxis Ltd. "We have worked with patients, clinicians and patient organisations in order to best respond to NICE's questions during this approval process, and we will continue to work alongside NICE and the CF community to ensure all eligible patients within this positive recommendation can benefit from treatment".

For all other enquiries relating to Bronchitol or Pharmaxis, please contact our UK-based office at eu.info@pharmaxis.com

About Pharmaxis

Pharmaxis (ACN 082 811 630) is a specialist pharmaceutical company involved in the research, development and commercialisation of therapeutic products for chronic respiratory disorders. Its product Aridol® for the assessment of asthma is launched in a number of key markets.  Its development pipeline of products includes: Bronchitol for CF, bronchiectasis and chronic obstructive pulmonary disease (COPD), ASM8 for asthma, PXS25 for idiopathic pulmonary fibrosis and a new oxidase inhibitor for lung disease.  Pharmaxis is listed on the Australian Securities Exchange (symbol PXS). The company is headquartered in Sydney at its TGA-approved manufacturing facilities. For more information about Pharmaxis, go to http://www.pharmaxis.com.au.

About Bronchitol

Bronchitol has been developed to help clear mucus (a major source of lung infections), improve lung function and reduce exacerbations in patients with CF. 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 in treating patients with CF.

About Cystic Fibrosis

In a healthy person, there is a constant flow of mucus over the surfaces of the air passages in the lungs, removing debris and bacteria. In CF, an inherited disease, a defective gene disrupts ion transport across the epithelial membrane within cells. In the lungs, this leads to a depletion of the airway surface liquid that normally bathes the cilia, and a resultant reduction in mucociliary clearance. The result is thick, sticky mucus that clogs the lungs, severely restricting the natural airway-clearing process. It also increases the potential for bacteria to become trapped causing inflammation within the airway. This unhealthy lung environment can lead to life-threatening lung infections and symptom exacerbations that reduce respiratory function and that may result in the need for lung transplantation and/or premature death.

About the National Institute for Health and Clinical Excellence (NICE)^[2]

NICE was set up in the UK in 1999 to reduce variation in the availability and quality of NHS treatments and care - the so called 'postcode lottery'. The evidence-based guidance and other products help resolve uncertainty about which medicines, treatments, procedures and devices represent the best quality care and which offer the best value for money for the NHS. The organisation also produces public health guidance recommending best ways to encourage healthy living, promote wellbeing and prevent disease. This public health guidance is for local authorities within England and Wales, the NHS and all those with a remit for improving people's health in the public, private, community and voluntary sectors.

Every piece of NICE guidance and every NICE quality standard is developed by an independent committee of experts including clinicians, patients, carers and health economists. All guidance is considered and approved by the NICE Guidance Executive, a committee made up of NICE executive directors, guidance centre directors and the communications director, prior to publication.

Forward-Looking Statements

Forward-looking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential for Aridol/Osmohale and/or Bronchitol. All forward-looking statements included in this media release are based upon information available to us as of the date hereof, and we assume no obligation to update any such forward-looking statement as a result of new information, future events or otherwise. We cannot guarantee that any product candidate will receive regulatory approval or that we will seek any such approval.

References

1.http://www.cftrust.org.uk/aboutcf/publications/cfregistryreports/ (Annual Data Report 2010)

2.http://www.nice.org.uk/aboutnice/whoweare/who_we_are.jsp. Accessed from the NICE website on 1.10.12

Contacts: For further information about this press release, please contact Alex Pugsley at Chandler Chicco Agency on +44-(0)207-632-1889.