Novartis announced it won U.S. FDA approval for its experimental sickle cell disease drug, Adakveo, making it the first of several proposed new therapies designed to offer relief for patients with the debilitating blood disease to get U.S. regulatory clearance.
The biologic was shown in trials to cut sickle cell pain crises nearly by half — from 2.98 incidents annually in those getting a placebo to 1.63 incidents annually.
According to the drugmaker, Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin — a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.
The medicine is expected to be available to patients in a matter of weeks.
Read the press release
