Medication for treating CF

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NICE Approves Pharmaxis’ Bronchitol® As An Important New Treatment Option for Cystic Fibrosis Patients

The European headquarters of Australian pharmaceutical company Pharmaxis (ASX: PXS) 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 Fibrosisi 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 4,500 adults. Patients with CF can experience a rapid decline in lung function, with frequent respiratory infections (exacerbations) that can require hospitalisation.


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 28th, 2012, subject to any appeals or factual corrections received by NICE. See more information on the 28th by clicking


“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”.


Buckinghamshire, UK – 26th October 2012

Source: CF Worldwide


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