FDA OKs Adakveo, first targeted therapy for painful complication of sickle cell disease

The US Food and Drug Administration on Friday approved Adakveo (crizanlizumab-tmca), a treatment to reduce the frequency of vaso-occlusive crisis – a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells – for patients aged 16 years and older.

Developed by Swiss pharma giant Novartis (NOVN: VX), Adakveo, which was accepted for priority review by the FDA in July this year, 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, and marks a new era in the treatment of sickle cell disease.

Novartis' shares edged up 1.1% in early trading today.