FDA approved crizanlizumab-tmca, a monoclonal antibody against the P-selectin glycoprotein that is expressed on activated endothelial cells and platelets, to reduce the frequency of vaso-occlusive crisis in patients aged 16 years and older with sickle cell disease (SCD).
It is the first targeted therapy approved for sickle cell disease.
Approved in a randomized clinical trial of 198 patients with SCD and a history of vaso-occlusive crisis.
Patients receiving crizanlizumab averaged 1.63 pain episodes per year, and 35% had no episodes, while placebo group averaged 2.98 pain episodes and 17% had no episodes.
Crizanlizumab treatment also delayed the time to first vaso-occlusive crisis from 1.4 months to 4.1 months.
It specifically inhibits selectin, a substance that contributes to red blood cells sticking together that leads to vaso-occlusive crisis.
Adverse events with crizanlizumab: include back pain, fever, joint pain, and nausea. Patients are monitored for infusion-related reactions, and for interference with automated platelet counts or platelet clumping.
The list price for the monthly infusion will be approximately $85,000 to $113,000 per year.