NICE Approves Pharmaxis' Bronchitol® As An Important New Treatment
Option For Cystic Fibrosis Patients
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
28th, 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.
