What is 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.
What effect does Kalydeco 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.
Is Kalydeco available in the UK?
Kalydeco is available for use in the UK for people with CF over the age of six months with one of nine rare gating mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G139D), and more recently the R117H mutation has receive expanded access for those under 18.
On 18 September 2020, the EMA announced they had adopted a positive opinion to expand the eligibility of Kalydeco to babies from the age of four months. It is expected to take up to two months for the European Commission to formally grant this licence extension, after which clinicians will be able to prescribe Kalydeco according to this expansion.
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