Vertex Pharmaceuticals is a biotech company focused on developing treatments for cystic fibrosis. The company’s approved CF drugs include Kalydeco, Orkambi, Symdeko and Trikafta. In December, the Food and Drug Administration approved Vertex’s sickle cell disease treatment Casgevy, the world’s first approved therapy using CRISPR gene editing technology. Vertex’s pipeline also includes non-CF treatment candidates such as exa-cel for beta-thalassemia, VX-147 for APOL1-mediated kidney disease and nonopioid drugs for pain treatment.
Vertex reported $2.48 billion in product revenue in the third quarter, up 6% year over year. Vertex’s Trikafta-Kaftrio triple combination therapy for CF accounted for about 92% of the company’s total sales in the third quarter.
Looking ahead, Vertex anticipates several new product commercial launches, including Casgevy, exa-cel for treating transfusion-dependent beta-thalassemia, VX-548 for treating acute pain, and a combination of vanzacaftor, tezacaftor and deutivacaftor for treating CF.