The US Food and Drug Administration has granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).
The drug is under development by Swiss pharma giant Novartis (NOVN: VX), which has previously said it plans regulatory filings this year.
Also known as sickle cell pain crises, VOCs are unpredictable and extremely painful events that can lead to serious acute and chronic complications. VOCs happen when multiple blood cells stick to each other and to blood vessels, causing blockages. Treatments that make blood cells and blood vessels less sticky may help reduce the number of days patients experience VOCs.
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