Boston-based Vertex Pharmaceuticals Inc. has achieved one of the biggest feats to date for cystic fibrosis patients — a treatment that could benefit 90 percent of patients with the rare lung disease.
The FDA approved a combination of three drugs Monday called Trikafta, which targets a mutation in the F508del gene. That genetic error is found in approximately 90 percent of patients with the rare lung disease.
"If you think about it, we went from being able to treat no patients to being able to treat nearly all patients in seven years. That's never been done in the industry, to my knowledge," longtime CEO Jeff Leiden told the Business Journal.