The Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for elexacaftor (VX-445), tezacaftor and ivacaftor triple combination regimen for patients with cystic fibrosis.
Elexacaftor is an investigational therapy, while the combination of tezacaftor and ivacaftor and ivacaftor alone are both already approved treatments for cystic fibrosis. The NDA submission includes data from two phase 3 trials assessing the triple combination therapy in patients with one F508del mutation and one minimal function mutation, as well as those with two F508del mutations.
Results from both studies showed that treatment with elexacaftor, tezacaftor and ivacaftor led to statistically significant improvements in lung function based on percent predicted forced expiratory volume in one second (ppFEV1).
“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,” said Reshma Kewalramani, MD, Executive Vice President and Chief Medical Officer at Vertex.
The FDA has set a Prescription Drug User Fee Act (PDUFA) date of March 19, 2020 for this application.
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