Press release
Vertex Announces FDA Acceptance of New Drug Application for Vanzacaftor/Tezacaftor/Deutivacaftor, a Next-In-Class Triple Combination Treatment for Cystic Fibrosis
- Vanza triple granted priority review with Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025 – - EU Marketing Authorization
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Vanza triple granted priority review with Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025 –\n\n\n- EU Marketing Authorization Application (MAA) submission also validated by European Medicines Agency (EMA)\n\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy (vanza triple) for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to the vanza triple. Vertex used a priority review voucher for this submission reducing the review time from 10 months to 6 months, resulting in a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025.\n\n\n“The FDA acceptance of our vanza triple application and the MAA validation by the EMA represent important milestones in the decades-long development of CFTR modulators and another example of our track record of serial innovation in CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex. “Vanzacaftor raises the high bar set by TRIKAFTA® and gives more people with CF the chance to get to levels of sweat chloride below the diagnostic threshold for CF, and even to levels of sweat chloride seen in those without CF.”\n\n\nVertex also received validation of its Marketing Authorization Application (MAA) submission by the European Medicines Agency (EMA) in the EU for patients ages 6 years and older. The company has also submitted in Canada, Australia, Switzerland and the U.K.\n\n\nAbout Cystic Fibrosis\n\n\nCystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many differen...