Novartis has secured its crizanlizumab (SEG101) Breakthrough Therapy designation US Food and Drug Administration (FDA), for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).
Painful sickle cell crises matter because they can disrupt patients’ lives, and often require hospital visits and medical attention, explained in a statement Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development. Novartis will push in the next period to have crizanlizumab, a therapy that has the potential to prevent sickle cell pain crises, available in the US as soon as possible, he added.
VOCs, also known as sickle cell pain crises, 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.
The Phase II SUSTAIN trial, which compared the P-selectin inhibitor crizanlizumab with placebo in patients with sickle cell disease, also showed that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo during treatment, Novartis said in its press release.
EDIT: There were typos in the title of this article.