FDA staff say Vertex CF combo works, unclear how
(Reuters) – Vertex Pharmaceuticals Inc's combination of an experimental compound and an approved drug significantly improved lung function in cystic fibrosis patients with the most common genetic mutation underlying the disease, FDA staff said. The U.S. Food and Drug Administration staff reviewers, however, were not sure whether Vertex's already approved therapy, Kalydeco, had a positive effect alone. The FDA is trying to ask the panel if the evidence is enough to show that the combination's benefit is significantly better than that of a single component, RBC Capital analyst Michael Yee said. The FDA staff was satisfied with the safety profile of the combination, to be called Orkambi, according to documents released on Friday.