New Sickle Cell Drug, ADAKVEO, Approved!
The U.S. Food and Drug Administration (FDA) approved Novartis’ Adakveo (crizanlizumab) to decrease the frequency of pain crises, in adults and children age 16 years and older with sickle cell disease. Novartis indicates it expects to make the therapy available in the coming weeks.
Sickle cell disease is a genetic condition that affects the body’s red blood cells. It occurs when a child receives two sickle cell genes—one from each parent. In someone living with this disease, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. This can lead to acute pain episodes called VOCs, in addition to life-threatening complications. Those life-threatening conditions include stroke, vision loss, blood clots in the lungs, and infections.
It is estimated that: