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Studies for Orkambi alternative show promising results

Two global Phase III trials using the compound tezacaftor in combination with ivacaftor (Kalydeco) have revealed its impact for people with various combinations of the cystic fibrosis (CF) gene.

The studies, published today in the New England Journal of Medicine, show the new combination therapy to have comparable effects to Orkambi (which combines ivacaftor with a compound called lumacaftor) for people with two copies of the F508del mutation, and significant improvements for those with one F508del and one ‘residual function’ mutation.

The EVOLVE study involving those with two copies of the F508 del mutation, presented in the journal by former Cystic Fibrosis Trust board member Professor Stuart Elborn, found that the tezacaftor/ivacaftor combination treatment was more stable than Orkambi, with similar benefits of around four per cent lung function improvement, and fewer side effects.

The EXPAND study, presented by Professor Jane Davies, found an average absolute improvement in FEV1 lung function of 6.8 per cent in those with one F508del and one residual function mutation, compared with the placebo.

The Cystic Fibrosis Trust first reported findings from these trials in March this year.

Next steps

Both studies involved people with CF age 12 and over. At the end of August the US Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) both accepted applications to consider licencing for the combination treatment for those age 12 and over with two copies of F508del or one F508del and a residual function mutation.

Both the FDA and EMA announced they would be prioritising the drug for consideration, with results expected in 2018.

Participating in trials is key

Dr Keith Brownlee, Director of Impact at the Cystic Fibrosis Trust, welcomed the two publications, and praised those who took part in these ground-breaking trials: “This is a very exciting time for people with CF and precision medicine. Phase III trials like these are critical, and they cannot happen unless people take part.

“That is why the Trust set up the Clinical Trials Accelerator Platform, to support access to CF clinical trials – and ultimately to help us stop the clock on cystic fibrosis.”

In 2017 the Trust launched its Clinical Trials Digital Hub and its very own Trials Tracker where people with CF can find trials available to them.

Explore the Hub

Professor Elborn, who is Clinical Lead for the Trials Accelerator centre at the Royal Brompton, said: “The combination of tezacaftor and ivacaftor used in these trials is an important step forward in the treatment of CF, and could set the scene for trials of a three-drug combination in the future. The Trust’s Clinical Trials Accelerator Platform will be a valuable resource in drug development like this, and we’re delighted to be participating in it.”

Triple combination therapy

Vertex also announced promising results this year from Phase I and II trials of variations of a brand new ‘triple combination therapy’, using a combination of ivacaftor, tezacaftor and one of three new compounds. Find out more.

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