Breakthrough Therapy Designation Awarded to Crizanlizumab, a New Treatment Option for Sickle Cell Disease

Cassandra Kuhn & Olivia Reiber

Abstract


Sickle cell disease is an inherited disease that affects hemoglobin of red blood cells. It can cause patients extreme pain due to the blockage of small blood vessels. These episodes of extreme pain are called vaso-occlusive crises, or pain crises, and they can lead to increased morbidity and mortality for patients with sickle cell disease. Crizanlizumab is a new therapy that recently received Breakthrough Therapy designation from the FDA. This medication helps reduce the amount of pain crises by blocking P-selectin on sickle cells from binding to one another, preventing the stasis of blood flow. This new therapy is unlike any treatment currently available and is very hopeful for patients with sickle cell disease.


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