Sickle cell infusion gains FDA breakthrough designation

The Food and Drug Administration has granted breakthrough therapy designation to crizanlizumab, a monthly infusion for the prevention of vasoocclusive crises in patients with sickle cell disease of all genotypes.

The designation allows the treatment to be reviewed on an expedited schedule.

Crizanlizumab, marketed by Novartis, is a humanized anti–P-selectin monoclonal antibody that has been shown to inhibit interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes.

In the phase 2 SUSTAIN trial, crizanlizumab reduced the median annual rate of vasoocclusive crises that resulted in health care visits by about 45%, compared with placebo (1.63 vs. 2.98; P = .010). The drug also increased the percentage of patients who did not experience any vasoocclusive crises, compared with placebo (35.8% vs. 16.9%; P = .010).

The rates of treatment-emergent and serious adverse events was similar in the drug and placebo arms of the trial.

mschneider@mdedge.com