Orkambi update, 21st November 2015
Overnight Orkambi, the drug combination of lumacaftor and ivacaftor received EU approval for people with CF 12+ with two copies of the F508del mutation.
The reimbursement process will now begin country by country. Great news for Europeans with CF and let's hope we get the same good news here in Australia in early 2016.
Breaking news, 19th November 2015
Overnight the European Commission approved the use of ivacaftor in children with cystic fibrosis ages 2 to 5 who have one of 9 gating mutations and people with cystic fibrosis ages 18 and older who have an R117H mutation. The Country-by-country reimbursement processes will now begin across Europe for each new indication.
In people with a gating mutation, or a R117H mutation, the CFTR protein reaches the cell surface but does not work properly.Known as a CFTR potentiator, Kalydeco (ivacaftor) is an oral medicine designed to help CFTR proteins at the cell surface open more often to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.
Life expectancy in Australia is 37 years however Cystic Fibrosis Australia is working to extend life expectancy to 50 years by 2025 through its clinical quality improvement programs, research and advocacy to ensure all people with CF have access to the best medicines and treatments.
CEO Update, 11th November 2015
For the latest communique from Cystic Fibrosis Australia CEO, Nettie Burke click here
Vertex has submitted Orkambi to the TGA and PBAC and it is hoped that following consumer input early in the new year and potentially some stakeholder meetings, approval will be granted in March 2016.
Orkambi has had mixed trial results for people with two F508del CFTR mutations however it is vital to note that many people saw a decrease in exacerbations, hospitalizations and lung damage and importantly in many cases people's quality of life improved.
Vertex has a compassionate access program for people on the Orkambi trial and others with severe disease. If you are interested in learning more about the Orkambi compassionate access program speak to your CF clinician.
Cystic Fibrosis Australia will continue its government advocacy and since the creation of CF CAN, our new consumer advocacy network, we now have well informed supporters to further enhance our message.
New Research Project
The Australian Cystic Fibrosis Research Trust (ACFRT) is pleased to announce funding of a new research project in conjunction with the University of Wollongong called 'Multi-action antibiotics to treat chronic biofilm infections'. For more...
Welcome to the CF Federation, Australia
Cystic Fibrosis (CF) is the most common, genetically acquired, life-shortening chronic illness affecting young Australians today.
It primarily affects the lungs and the digestive system, however, it also affects a number of other organs and systems.
Complications increase with age, requiring ever increasing levels of (self) care and support.
On average one in 25 people carry the CF gene (most of whom are unaware they are carriers).
To find out more about CF click ABOUT CF on the toolbar above.
Cystic Fibrosis support services organisations exist in every major jurisdiction of Australia, with the exception of the Northern Territory.
People living with CF in the Northern Territory are often supported by one or more of the adjoining CF organisations.
To contact your nearest CF organisation click CONTACT US on the toolbar above.
Most CF organisations in Australia began as member peer-support and self-help groups, growing over time to member services associations.
Today nearly all of these are modern not-for-profit health support services and research organisations involved with every aspect of living with
Cystic Fibrosis in their jurisdictions, including providing support services, raising public awareness, promoting carrier screening,
supporting research and lobbying for better health and wellbeing outcomes for those living with CF.
To find out more select the relevant entity on the map of Australia at top right of screen.
In 2003 Cystic Fibrosis Australia was incorporated as a company limited by guarantee with a number of the larger CF organisations as founding members and guarantors. Cystic Fibrosis Australia is focussed on national sponsorship, advocacy and awareness raising and on the promotion and funding of research.
All State and Territory CF organisations together with Cystic Fibrosis Australia work together collaboratively in a loosely coupled network to ensure better standards of care and quality of life for all Australians living with Cystic Fibrosis.
Funding for research into Cystic Fibrosis in Australia is through two principal foundations: the Australian Cystic Fibrosis Research Trust (ACFRT) and Cystic Fibrosis Research Limited. To learn more about CF research select RESEARCH on the toolbar above.