FDA Approval for Combination Drug Symdeko

In Australia, we woke to the news this morning that the U.S. Food and Drug Administration (FDA) in the USA (the equivalent of our Therapeutic Goods Adminstration or TGA in Australia) had approved the tezacaftor/ivacaftor combination drug Symdeko.

This is a drug that improves the function of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation or who have one of 26 specified mutations, even if they do not have a copy of the F508del mutation.

Late stage trials showed:

1. 4%-6.8% increase in lung function (FEV) (dependent on the mutation)
2. 35% decrease in exacerbations of the lung
3. Less side-effects than Orkambi (indirect comparison)


This is great news for the CF community in the USA but the question now arises, “When are Australians living with CF going to get access?”


The answer is complicated and by no means certain. The process to Pharmaceutical Benefits Scheme (PBS) listing is complex and there are many opportunities for new drugs to falter along the path to listing on the PBS. First however, the Therapeutics Goods Administration (TGA) in Australia have to approve it. Basically, the TGA determine that it is safe for use in humans. Vertex have Symdeko before the TGA right now.

Assuming that Symdeko gets the OK from the TGA in the first quarter of 2019 it could then take up to two years to get PBS listing approval through the Pharmaceutical Benefits Advisory Committee (PBAC). That’s 2021. It took two years to get Kalydeco listed on the PBS and Orkambi is still trying two years on. The system is clearly in need of an overhaul.

Could Symdeko leapfrog Orkambi and get listed sooner? The answer is probably not. That’s why it’s important to continue to lobby for the listing of Orkambi whilst Symdeko goes through to the TGA and PBAC process. Vertex feel that once the process for the listing of Orkambi has been established within the PBAC, this should provide a framework to navigate and accelerate the listing not only for Symdeko but also other triple combination therapies currently in the pipeline.

Two years is a long time to wait while lungs continue to deteriorate. The CF Federation here in Australia continue to work hard to bring pressure to bear on pharmaceutical companies, the PBAC and the Department of Health to ensure that people living in Australia can have access to life saving drugs as soon as possible.

We do not seek to compromise safety but are looking at every step of the process to see where we can help. For example at the clinical trial level, CFWA were able to provide seed funding to establish an adaptive clinical trial platform to reduce the amount of time patients spent on placebo and hopefully reduce the amount of time spent in clinical trials. See the latest edition of Red Magazine for more information.

You can learn more about Symdeko from the US CF Federation by clicking here and view Vertex’s press release here.


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