Information for the public
What has NICE said?
Lumacaftor–ivacaftor (Orkambi) is not recommended for treating cystic fibrosis in people 12 years and older who are homozygous for the F508del mutation.
Lumacaftor–ivacaftor should not normally be prescribed on the NHS for people with cystic fibrosis who have the F508del mutation. Your doctor should talk to you about other treatment options available to you.
If you have the F508del mutation and are already taking lumacaftor–ivacaftor for cystic fibrosis, you should be able to continue taking it until you and your doctor decide it is the right time to stop.
Cystic fibrosis is an inherited disease that affects the lungs by clogging them with thick and sticky mucus. This causes coughing, shortness of breath and increases the risk of lung infections.
People who are homozygous for the F508del mutation have 2 copies of a faulty gene, which results in cystic fibrosis.
Lumacaftor–ivacaftor helps to treat the cause of cystic fibrosis.
NHS Choices may be a good place to find out more.
These organisations can give you advice and support:
NICE is not responsible for the quality or accuracy of any information or advice provided by these organisations.