Vertex's New Drug Application for Cystic Fibrosis Treatment Accepted by FDA

Vertex Pharmaceuticals Inc. announced that the U.S. FDA has accepted its New Drug Application (NDA) for its investigational once-daily vanzacaftor/tezacaftor/deutivacaftor triple combination therapy, known as vanza triple, for cystic fibrosis (CF) patients aged six and older who have at least one F508del mutation or another responsive mutation in the CFTR gene. By using a priority review voucher, Vertex expedited the review process from 10 months to six months, setting a Prescription Drug User Fee Act (PDUFA) target action date of January 2, 2025.

Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex, highlighted that the FDA's acceptance and the European Medicines Agency's (EMA) validation of the MAA submission mark significant milestones in developing CFTR modulators. She emphasized that vanzacaftor aims to elevate the standards set by TRIKAFTA®, providing more individuals with CF the opportunity to reach sweat chloride levels below the diagnostic threshold for CF.

Vertex also submitted its MAA for vanza triple in the EU for patients aged six and older, with similar submissions in Canada, Australia, Switzerland, and the U.K.

Cystic fibrosis is a rare genetic disorder affecting over 92,000 individuals globally. It is a progressive disease impacting multiple organs, including the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive system. The disease is caused by mutations in the CFTR gene, leading to defective or missing CFTR protein. CFTR mutations disrupt the flow of salt and water in and out of cells, resulting in thick, sticky mucus in the lungs, chronic infections, and progressive lung damage. The median age of death for CF patients is in the 30s, but treatments are improving this prognosis.

Currently, Vertex's CF medicines treat over 65,000 people across 60 countries, representing two-thirds of diagnosed CF patients eligible for CFTR modulator therapy. CF diagnosis often involves genetic testing and assessing sweat chloride levels (SwCl), which reflect CFTR protein dysfunction. A SwCl level of 60 mmol/L or higher indicates CF, while levels between 30-59 mmol/L suggest possible CF, requiring further testing. Levels below 30 mmol/L are typical for CF carriers who do not manifest the disease. Lower SwCl levels are linked to improved outcomes, such as stable lung function and better quality of life.

The vanza triple combination includes vanzacaftor and tezacaftor, designed to increase CFTR protein at the cell surface, and deutivacaftor, a potentiator that enhances the channel open probability of the CFTR protein. The investigational therapy has received Fast Track and Orphan Drug Designations from the U.S. FDA for CF treatment.

Vertex is a global biotechnology company focused on creating transformative medicines for serious diseases. The company has approved treatments for cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia. Vertex is also advancing clinical programs in acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1, and alpha-1 antitrypsin deficiency.

Founded in 1989, Vertex's global headquarters is in Boston, with international headquarters in London. The company has R&D sites and commercial offices worldwide. Vertex has been recognized as a top employer in the industry, featuring on Science magazine's Top Employers list for 14 consecutive years and Fortune’s 100 Best Companies to Work For.