Breakthrough status for Novartis’ sickle cell disease therapy

Breakthrough sickle cell disease study could lead to new treatments
Credit: Kateryna Kon

US regulators have granted Novartis’ crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).

Also known as sickle cell pain crises, VOCs are unpredictable and extremely painful events that can lead to serious acute and chronic complications. They 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.

Breakthrough Therapy designation was granted based on positive results of the Phase II SUSTAIN trial, which compared the P-selectin inhibitor crizanlizumab with placebo in patients with sickle cell disease.

Samit Hirawat, Head of Novartis Oncology Global Drug Development, said: “We look forward to working closely with the FDA over the coming months toward making crizanlizumab, a therapy that has the potential to prevent sickle cell pain crises, available in the US as soon as possible.”