Orkambi - CF Buzz

Orkambi Update 1st June

Vertex sent a proposal to the Department of Health seeking a temporary access arrangement for Orkambi.




Orkambi Update 16th May

The PBAC ruling rejecting the reimbursement of Orkambi and the lack of support and interest in healthcare in the Federal Government’s budget have caused a great deal of disappointment and dissatisfaction in the cystic fibrosis community. Almost 3,500 Australians live with cystic fibrosis (CF) and more than 200,000 family members and carers provide vital support. They all dream about the development of a cure for CF. For more than a decade Vertex has been working on the development of CF modulator drugs.

The hopes of the CF community were raised when Vertex found success with Orkambi only to be dashed by a PBAC rejection. Two issues concern Cystic Fibrosis Australia (CFA) greatly. Firstly, hundreds of millions of dollars are invested into pharmaceutical research and development and life saving discoveries are held back from desperately ill Australians. Secondly, the current drug funding process is not designed to accommodate high cost personalised medicines. And there are many more in the pharmaceutical pipeline.

This issue is not going away. Cystic Fibrosis Australia is calling for a total overhaul of the system and the establishment of robust Phase Four Trials before this health liability results in avoidable deaths. For the past 18 years CFA has managed the Australian Cystic Fibrosis Data Registry and has the experience and expertise to manage ongoing review of the efficacy and safety of new drugs. CFA is adamant that both soft and hard measures need to be assessed and a “tele-health” based multidisciplinary team approach is required to support people as they begin their modulator drug therapy.

CFA’s CEO Nettie Burke said today that, “CFA’s primary concern is access. Australians need access to life saving drugs. Cost are reportedly high and in some instances obscenely high and so robust post market evaluation trials needs to be established”. Drug companies should receive realistic reimbursement when a drug has positive outcomes for patients. Equally, if the outcomes are non-existent, patients should be encouraged and supported to undertake a new trial or treatment. CFA is currently developing a Clinical Trial Portal that will provide international and local trial notifications and protocols to the general public. People will now know what is happening in CF innovations across the world and have the opportunity to discuss options with their clinicians.

Read original transcript on Cystic Fibrosis Australia’s website.


Orkambi Update 21st November

Overnight Orkambi, the drug combination of lumacaftor and ivacaftor received EU approval for people with cystic fibrosis (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.


Orkambi Update 19th November

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. 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.

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.

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.


Orkambi Update 11th November

For the latest communique from Cystic Fibrosis Australia CEO, Nettie Burke click here Orkambi Communique.

Vertex has submitted Orkambi to the TGA and PBAC and it is hoped that following consumer input 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 exacerbation, hospitalisation and lung damage and importantly in many cases quality of life was 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.



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