Most people have heard of cystic fibrosis, even if they have never met anyone who lives with it. It is an inherited illness that clogs the lungs with thick mucus and makes every breath harder. In the United States and across much of Europe, most of the people treated for it now take tablets every day that help their lungs work better. Those tablets, in most cases, come from Vertex Pharmaceuticals.
A success like this is rare in medicine, and it has made Vertex a large and wealthy company. It has also tied Vertex to a single disease. In recent years the company has started selling medicines for very different conditions: a gene-editing treatment for inherited blood disorders, a pain medicine that is not an opioid, a drug for a rare hormonal illness. Can Vertex become more than a cystic fibrosis company?