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Novartis drug for sickle cell disease gets breakthrough status from FDA

Switzerland's Novartis AG said the U.S. Food and Drug Administration granted its investigational drug crizanlizumab, or SEG101, breakthrough-therapy status.

The designation covers the prevention of vaso-occlusive crises, or VOCs — a common, painful complication of sickle cell disease, linked to increased morbidity and mortality. VOCs occur when multiple blood cells stick to each other and to blood vessels, causing blockages.

Breakthrough-therapy status expedites the development and review of drugs meant to treat serious conditions.

The designation was based on data from a mid-stage trial, called Sustain, which showed crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3% compared to placebo. Also, significantly more patients receiving crizanlizumab did not experience VOCs: 35.8% versus 16.9% in the placebo group.

Novartis said it plans to file for crizanlizumab's FDA approval in the first half of 2019.

Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder typically have distorted, crescent red blood cells, shaped like sickles. Patients experience pain when these stiff and inflexible red blood cells get stuck in small blood vessels. A particular complication of sickle cell disease is high blood pressure in the blood vessels that supply the lungs.