Vertex has selected the triple combination of VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential regulatory approvals for people ages 12 and older with cystic fibrosis.

Vertex Pharmaceuticals Incorporated announced that it has selected the triple combination of the next-generation corrector VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential global regulatory approvals for people ages 12 and older with cystic fibrosis (CF).

Final data announced from a 24-week Phase III study in people with one F508del mutation and one minimal function mutation and from a 4-week Phase III study in people with two F508del mutations will form the basis of these submissions. Vertex previously announced that both of these Phase III studies met their primary endpoints, and the company announced the final results of these studies, including results for key secondary endpoints and safety data. In each study, treatment with the VX-445 triple combination regimen resulted in statistically significant improvements in all key secondary endpoints.

Data from the 24-week study in people with one F508del mutation and one minimal function mutation showed a mean absolute improvement in percent predicted forced expiratory volume in one second (ppFEV1) of 14.3 percentage points from baseline (p<0.0001) and a 63 reduction in the annualized rate of pulmonary exacerbations p><0.0001) through week 24 in patients who received the vx-445 triple combination regimen compared to those who received triple placebo. the vx-445 triple combination regimen was generally well tolerated across the two phase iii studies. in the study in people with one f508del mutation and one minimal function mutation 2 and 0 patients respectively who received the vx-445 triple combination or triple placebo discontinued treatment due to adverse events. there were no discontinuations for adverse events in either arm of the study in people with two f508del mutations.>

Vertex plans to submit a New Drug Application (NDA) to the FDA in the third quarter of 2019 and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fourth quarter of 2019 based on these data in people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation and in people with two F508del mutations.