Information for the public
Ivacaftor–tezacaftor–elexacaftor
Ivacaftor–tezacaftor–elexacaftor (Kaftrio), taken with ivacaftor (Kalydeco), is available on the NHS. It is a possible treatment for cystic fibrosis in people 2 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Tezacaftor–ivacaftor
Tezacaftor–ivacaftor (Symkevi), taken with ivacaftor, is available on the NHS as a possible treatment for cystic fibrosis in people 6 years and over who have:
- 2 copies of the CFTR gene with F508del mutations, or
- a copy of the CFTR gene with an F508del mutation and a copy of the CFTR gene with 1 of the mutations listed in section 2.2 of the guidance.
Lumacaftor–ivacaftor
Lumacaftor–ivacaftor (Orkambi) is available on the NHS as a possible treatment for cystic fibrosis in people 1 year and over who have 2 copies of the CFTR gene with F508del mutations.
Is this treatment right for me?
Your healthcare professionals should give you clear information, talk with you about your options and listen carefully to your views and concerns. Your family can be involved too, if you wish. See our webpage on making decisions about your care.
Questions to think about
- How well does it work compared with other treatments?
- What are the risks or side effects? How likely are they?
- How will the treatment affect my day-to-day life?
- What happens if the treatment does not work?
- What happens if I do not want to have treatment? Are there other treatments available?
Information and support
The NHS webpage on cystic fibrosis may be a good place to find out more.
These organisations can give you advice and support:
- Cystic Fibrosis Trust, 0300 373 1000
- CF Voices, info@cfvoices.org
- Quest for a CF Cure, questforacure@gmail.com
You can also get support from your local Healthwatch.
NICE is not responsible for the quality or accuracy of any information or advice provided by these organisations.
ISBN: 978-1-4731-6250-1
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