Vertex Phase III For CF Combo Shows Power Of “Breakthrough” Designation
This article was originally published in Pharmaceutical Approvals Monthly
Pivotal trials for the combination of Kalydeco and VX-809 will measure safety and efficacy over 24 weeks, whereas Kalydeco monotherapy gained FDA approval on the basis of 56 weeks of data; Vertex and FDA developed the protocol together following a recent “breakthrough therapy” designation for the two-drug combo.
You may also be interested in...
Vertex/FDA in discussions on “breakthrough therapies”; Ipsen earnings hurting in EU; Pfizer discusses biosimilars; Krka likes emerging markets; Novo sees GLP-1 market competition stiffening
Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.
No device-related warning letters were released by the US FDA the week of 22 September.