The FDA acceptance is based on the previously announced positive results of two global Phase 3 studies in people with cystic fibrosis (CF)
Image: US FDA has accepted Vertex’s New Drug Application for triple combination regimen. Photo: Courtesy of The U.S. Food and Drug Administration/Wikipedia.
The US Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) of Vertex Pharmaceuticals for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen.
The biopharmaceutical firm said that the regulator has approved the priority review of the NDA and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of 19 March 2020.
Vertex executive vice president and chief medical officer Reshma Kewalramani said: “If approved, the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen would be a significant advance in CF treatment as the first CFTR modulator for those with one F508del mutation and one minimal function mutation, and bring additional benefit to patients with two F508del mutations.
“Our goal is to provide medicines that treat the underlying cause of CF to the vast majority of people with CF. We share a sense of urgency with people with CF, caregivers and clinicians to rapidly deliver innovative CF medicines to those waiting, and we look forward to working with the agency as they review the application over the course of the coming months.”
Cystic Fibrosis affects lungs, liver, GI tract, sinus, sweat gland, pancreas and reproductive tract
The FDA acceptance is based on the previously announced positive results of two global Phase 3 studies in people with cystic fibrosis, a 24-week Phase 3 study in people with one F508del mutation and one minimal function mutation and a 4-week Phase 3 study in people with two F508del mutations.
In addition, the triple combination regimen was generally well tolerated in both the Phase 3 studies that showed statistically significant improvements in lung function, which was the primary endpoint in all key secondary endpoints.
According to Vertex, CF is a rare, life-shortening genetic disorder that affects approximately 75,000 people across the globe. It is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinus, sweat gland, pancreas and reproductive tract.
CF is caused by a defective or missing CFTR protein, due to certain mutations in the CFTR gene, and is inherited to children who acquire two defective CFTR genes one from each parent.