Vertex Pharmaceuticals Incorporated is focused on the discovery, development, and commercialization of small molecule drugs targeting serious diseases. The company's main area of focus is cystic fibrosis. The company's lead marketed products are Trikafta, Symdeko/Symkevi, Orkambi and Kalydeco, which are collectively approved to treat the people with CF in North America, Europe and Australia. Trikafta, Vertex's triple combination regimen, was approved by the FDA for the treatment of CF in people aged 12 years and older who have at least one F508del mutation. While CF remains the main area of focus, Vertex is also developing treatments for sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, type 1 diabetes, pain, alpha-1 antitrypsin deficiency and muscular dystrophies. Pimodivir/VX-787, for the treatment of influenza, was out-licensed to Janssen while oncology candidates VX-970, VX-984 and VX-803 were divested to Merck KGaA....
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