Breakthrough made on combination treatment for cystic fibrosis

A combination therapy for cystic fibrosis will now progress to a phase III study.

There has been a new breakthrough on a combination treatment for patients with cystic fibrosis, it has been announced today (April 19th).
Vertex revealed that this new data suggests statistically significant improvements in lung function in sufferers with two copies of the F508del mutation after being treated with both the compound VX-661 and Ivacaftor.
The company now plans to run two 24-week phase III studies to support approval of the combination treatment for cystic fibrosis patients aged 12 and above.
Pending the results of the safety and efficacy data, Vertex would then submit a new drug application to the US Food and Drug Administration.
This would coincide with a Marketing Authorisation Application to the European Medicines Agency.
Chief executive of the Cystic Fibrosis Trust Ed Owen commented: "This is yet a further example of the major developments taking place in cystic fibrosis research."
As yet, there is no cure for this life-threatening genetic disease, with around 70,000 people worldwide estimated to have the condition.