The EVOLVE trial tested the new treatment in people with cystic fibrosis aged 12 and over with two copies of the F508del mutation, while the EXPAND trial looked at those with one copy of the F508del mutation and another mutation that leaves some residual function of the CFTR channel, which controls the movement of salt and water between cells.
The clinical trial results
In the EVOLVE group the drug improved lung function (FEV1) by an average of 4% and reduced exacerbations by 35% when compared with a placebo. In the EXPAND trial it was shown to improve FEV1 by 6.8% compared with the placebo group. Scientists described the drug as having a ‘favourable safety profile’, meaning that there were relatively few adverse effects on any of the participants and the drug was generally well tolerated.
What next for the new treatment?
These results show that the combination therapy is a promising potential treatment for some people cystic fibrosis. Vertex plans to submit a new drug application (in the USA) and a marketing authorisation application (in Europe) later this year. However, the processes involved in getting a new drug to market are long and complicated so it could be years before it is available, if it succeeds in passing all of the necessary checks along the way.
James Barrow, Head of External Affairs at the Trust, said: “These results represent reaching an important milestone on the path towards a future in which everyone living with cystic fibrosis has access to treatments that tackle the underlying cause of the condition. It's vital that we continue to work with the pharmaceutical industry and NHS to find a fair and sustainable model of reimbursement for new treatments, so that everyone has the opportunity to benefit.
Stopping the clock on cystic fibrosis
Today’s announcement reflects the exciting pipeline of new therapies being developed for cystic fibrosis. The Cystic Fibrosis Trust is committed to stopping the clock on cystic fibrosis by supporting this pipeline every step of the way, from clinical trials for new drugs to access on the NHS for those who could benefit.