International study published in Lancet shows that Orkambi improves the lung function of children with CF in less than 2 weeks.
24 July 2017
The international study, which included researchers from Imperial College London at the Royal Brompton Hospital, showed that Orkambi can improve the lung damage caused by the genetic disease in under two weeks.
The findings of the phase 3 trial involving 204 patients aged 6 to 11 years old were recently published in The Lancet Respiratory Medicine. Half the patients received Orkambi for six months, while the other half received a placebo.
At the end of the treatment period, researchers found two key improvements in markers of health amongst those who received the drug. Firstly, the children’s airways were significantly healthier based on a sensitive test of gas mixing in the lungs. Secondly, there was a 20 per cent reduction in the amount of chloride in their sweat – a common symptom of the disease.
Lead author Professor Felix Ratjen, of The Hospital for Sick Children at the University of Toronto, told the New Scientist: “We’ve shown that even in younger patients who have relatively mild disease, Orkambi led to improvements in lung function.”
Unlike Ireland, Orkambi is not yet available on the UK under the National Health Service.
Cystic fibrosis (CF) is caused by a defect in the gene cystic fibrosis transmembrane conductance regulator (CFTR), which codes for a protein that maintains the balance of salt and water in and out of cells. This leads to a build-up of thick and sticky mucus in the lungs and digestive system. Orkambi is one of the first therapies to target the commonest form of the underlying faulty CFTR protein rather than the symptoms.