Vertex Faces Choice After Second CF Drug Combo Succeeds in Phase 3

▤ Summary

An experimental Vertex Pharmaceuticals cystic fibrosis treatment that combines three of the company’s drugs has hit the main goals of two late-stage studies. CF is a genetic disorder caused by mutations to the gene that produces a protein that helps pump water in and out of cells. Without that protein, thick mucus builds up in the lungs, pancreas, and other organs, leading to breathing problems and bacterial infections. The Phase 3 studies tested this drug cocktail in two groups of CF patients with different genetic mutations. Vertex has been testing combinations of its drugs in an effort to expand the number of CF patients who can be treated.

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