Vertex (VRTX -0.57%), which makes multiple cystic fibrosis (CF) drugs, enjoys a monopoly over sales of treatments for the underlying cause of CF. Its dominance in CF was bolstered by the December 2024 approval of Alyftrek.
CF isn't Vertex's only market now, though. The big biotech company is ramping up commercialization efforts for Casgevy, a gene-editing therapy approved to treat two rare blood disorders -- sickle cell disease and transfusion-dependent beta-thalassemia.
Vertex also won FDA approval in January 2025 for Journavx (suzetrigine). This non-opioid drug is the first new type of pain medication approved in more than 20 years.
The company awaits accelerated approval for povetacicept in treating chronic kidney disorder IgA nephropathy. Vertex's pipeline also includes other promising candidates, including inaxaplin, which targets APOL1-mediated kidney disease.