Trikafta, made by Vertex Pharmaceuticals, is, as its name suggests, a combination of three drugs. It works for 90 percent of patients with cystic fibrosis, and will be priced at $311,000 per year.

Neither Trikafta nor Alfytrek address NBD1 ... desire for more treatment options for CF patients, support for a new mechanism of action that could provide clinically meaningful benefit for ...