Kalydeco

Ivacaftor, known by its brand name Kalydeco, was the first precision medicine for cystic fibrosis to become available on the NHS in December 2016.

Kalydeco is effective in anyone with at least one of the following nine gating mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D, or those with the R117H mutation, which around 5% of the population of people with CF in the UK have.

Outcomes calculated using data from the UK Cystic Fibrosis Registry show that median FEV1 (forced expiratory volume – a measure of lung function) increased by an average of 8.7% a significant improvement. People with CF often have higher levels of chloride in their sweat; tests on 439 people showed that their sweat chloride levels dropped by almost 50% on average after taking Kalydeco.

Kalydeco is available for use in the UK for people with CF over the age of four months with one of nine rare gating mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D) and over the age of 6 months for people with the R117H mutation.

For some rare mutations, Kalydeco is also available off-label on the NHS. You can find a list of these mutations here.