Approval of first targeted sickle cell therapy heralds arrival of new agents

Scoring approval about two months ahead of its PDUFA date, Adakveo crizanlizumab-tmca from Novartis AG (NYSE:NVS; SIX:NOVN) is the first targeted sickle cell therapy approved by FDA, designed to cut the disease’s pathology at the root.

The agency approved the anti-P selectin antibody to reduce the frequency of vaso-occlusive crises in patients 16 years and older with sickle cell disease. VOCs are painful episodes caused by blocked blood flow. ...