Vertex Pharmaceuticals

Vertex has transformed the treatment of cystic fibrosis with four approved drugs—Kalydeco, Orkambi, Symdeko/Symkevi, and Trikafta/Kaftrio—targeting different CFTR mutations. The company has expanded into genetic diseases like sickle cell disease and beta thalassemia through partnerships in gene editing/cell therapy. In January 2025 it received FDA approval for Journavx (suzetrigine) for acute pain in adults. Its pipeline also includes programs in type 1 diabetes using stem-cell derived therapies and additional genetic diseases. Clinical results have shown significant improvements in lung function for CF patients and promising early data in other indications.