Marketing Authorisation Application validated for cystic fibrosis treatment

The European Medicines Agency (EMA) has validated a Marketing Authorisation Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen, Vertex Pharmaceuticals (Europe) Limited has announced. 

The application was supported by positive results from two global Phase III studies in people with cystic fibrosis aged 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations.

“Today marks a significant milestone towards our efforts to bring new medicines to more people around the world who are living with cystic fibrosis,” said Reshma Kewalramani, MD, Executive Vice President and Chief Medical Officer at Vertex.

The two Phase III studies in people with cystic fibrosis (CF) consisted of a 24-week study in people with one F508del mutation and one minimal function mutation and a four-week study in people with two F508del mutations.

Both Phase III studies showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In these studies, the triple combination regimen was generally well tolerated.

“We are looking forward to working with the EMA on this important application,” Kewalramani said.