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' (a group that currently has no access to an effective), and people with two copies of the F508del mutation, and both trials produced positive results.
When will it become available?
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. Approximately 90% of the CF population in the UK have one copy of the F508del mutation, though it is expected that when the drug is first licensed, as in the United States, it will be available only to those over the age of 12. This accounts for 61% of the UK CF population. It is expected that at a later date, following completion of clinical trials in younger age groups, the drug will also be available to those under the age of 12.
Vertex has submitted the drug to the European Medicines Agency (EMA), who is analysing it for safety and clinical effectiveness. A decision is expected at the end of 2020.
If the EMA make a positive decision, Vertex Pharmaceuticals will be required to submit the drug to the National Institute for Health and Care Excellence (NICE), who will decide whether the drug will be licensed for use on the NHS.
In February, NICE announced its planned timelines for assessing the triple combination therapy, with 16 December 2020 the date they expect to publish their recommendation.
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What effect does the triple combination therapy have?
In clinical trials of the triple combination therapy, people with two copies of the F508del mutation had a 10% increase in lung function compared to treatment with Symkevi, and people with a single copy of F508del had more than a 14% increase in lung function compared to treatment with the placebo.
What are we doing about it?
We will continue to support the evaluation process, engaging all parties to work constructively to ensure that the triple combination therapy can be made available to people with cystic fibrosis as soon as possible.
Are there other therapies like this being developed?
The first triple combination therapy (brand name Trikafta in the US) has been submitted to the EMA. However, at least three companies, including AbbVie, 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.
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