What is Vertex's triple combination therapy?
The triple combination therapy (brand name Trikafta in the United States) uses a combination of ivacaftor, tezacaftor and elexacaftor. The drug was tested in clinical trials on two groups: people with one copy of the F508del mutation and one copy of a 'minimal function mutation', and people with two copies of the F508del mutation, and both trials produced positive results. In the United States, a licence has been granted by the Food and Drug Administration (FDA) for anyone over the age of 12 with one copy of F508del, regardless of their second mutation. This accounts for approximately 90% of the CF population.
When will it become available?
Recently, the triple combination therapy was approved in the United States for those aged 12 and over who have at least one F508del mutation. Vertex has submitted the drug to the European Medicines Agency (EMA), who is analysing it for safety and clinical effectiveness, with further updates expected in 2020. We will continue to support the evaluation process, engaging wherever possible, to ensure that the triple combination therapy can be made available to people with cystic fibrosis as soon as possible.
What can I do?
In the lead up to the 2019 general election we're asking you to contact your parliamentary candidates to ask them to commit to our four pledges to support people with cystic fibrosis, one of which is for the UK to lead the world in access to transformative new CF medicines like the triple combination therapy.
Are there other triple combination therapies?
Trikafta is the first triple combination therapy to be submitted to the EMA. However, at least three companies, including Galapagos Therapeutics, Proteostasis Therapeutics and Vertex Pharmaceuticals, are developing other potential triple combination therapies.
You can play a part in bringing the next generation of CF treatments to the people who stand to benefit from them by taking part in a clinical trial. Find out what trials might be right for you on our Trials Tracker.