For those of you who didn’t see the news a few weeks ago, On 30th May Vertex Pharmaceuticals announced that it has selected the triple combination of the next-generation corrector VX-445 (elexacaftor), tezacaftor and ivacaftor to submit for potential global regulatory approvals for people ages 12 and older with cystic fibrosis (CF). Final data announced today from a 24-week Phase 3 study in people with one F508del mutation and one minimal function mutation and from a 4-week Phase 3 study in people with two F508del mutations will form the basis of these submissions. Vertex previously announced that both of these Phase 3 studies met their primary endpoints, and the company today announced the final results of these studies, including results for key secondary endpoints and safety data.
Great news.