FDA News

First triple combination therapy for cystic fibrosis submitted to FDA for review

A new drug application for a triple combination regimen of elexacaftor plus tezacaftor/ivacaftor has been submitted to the FDA, with a request for priority review, according to a manufacturer press release.

Vertex, the manufacturer of elexacaftor (VX-445) and tezacaftor/ivacaftor (Symdeko), based the submission on the positive results of two global phase 3 studies, including a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations.

In both studies, the triple combination therapy was associated with statistically significant improvements in the primary endpoint of lung function, as defined by percent predicted FEV1, and key secondary endpoints. The triple combination therapy was also well tolerated, according to the release.

The triple combination regimen of elexacaftor plus tezacaftor/ivacaftor was granted breakthrough therapy designation in May 2018 in the United States.

“We have relentlessly focused on the progression of VX-445 (elexacaftor), tezacaftor and ivacaftor from discovery through clinical development and regulatory submission,” Reshma Kewalramani, MD, executive vice president and chief medical officer at Vertex, said in the release. “The submission of the NDA is a major step toward our goal of bringing this medicine to the largest remaining group of people with [cystic fibrosis] that still do not have an approved Vertex medicine, as well as toward providing significantly enhanced benefits to patients with two F508del mutations. We will continue working with the FDA as they review the NDA and look forward to the potential of this triple combination regimen becoming a new treatment option for people with [cystic fibrosis].”

A new drug application for a triple combination regimen of elexacaftor plus tezacaftor/ivacaftor has been submitted to the FDA, with a request for priority review, according to a manufacturer press release.

Vertex, the manufacturer of elexacaftor (VX-445) and tezacaftor/ivacaftor (Symdeko), based the submission on the positive results of two global phase 3 studies, including a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations.

In both studies, the triple combination therapy was associated with statistically significant improvements in the primary endpoint of lung function, as defined by percent predicted FEV1, and key secondary endpoints. The triple combination therapy was also well tolerated, according to the release.

The triple combination regimen of elexacaftor plus tezacaftor/ivacaftor was granted breakthrough therapy designation in May 2018 in the United States.

“We have relentlessly focused on the progression of VX-445 (elexacaftor), tezacaftor and ivacaftor from discovery through clinical development and regulatory submission,” Reshma Kewalramani, MD, executive vice president and chief medical officer at Vertex, said in the release. “The submission of the NDA is a major step toward our goal of bringing this medicine to the largest remaining group of people with [cystic fibrosis] that still do not have an approved Vertex medicine, as well as toward providing significantly enhanced benefits to patients with two F508del mutations. We will continue working with the FDA as they review the NDA and look forward to the potential of this triple combination regimen becoming a new treatment option for people with [cystic fibrosis].”