Crizanlizumab

Crizanlizumab is a monoclonal antibody developed by Novartis targeted towards P-selectin. It was announced by the company as an effective drug to prevent vaso-occlusive crisis in patients with Sickle cell anemia. The result of the Phase II SUSTAIN clinical trial was published on December 3, 2016 which showed the efficacy of the drug in sickle cell patients. Trial was conducted among 196 patients over 12 months which showed reduction in annual rate of sickle cell crises by 45% compared to the placebo group.[1]

Pathophysiology

P-selectin molecules are present on the surface of vascular endothelial cells and have been linked to Sickle cell vaso-occlusive crises.[2] Crizanlizumab selectively binds to these molecules and down regulates them to prevent the episodes.

References

  1. ↑ "Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease". New England Journal Medicine. Retrieved 5 December 2016.
  2. ↑ "Vaso-occlusion in sickle cell disease: pathophysiology and novel targeted therapies" (122(24)). 2013: 3892–3898.
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