Novartis has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for crizanlizumab (SEG101) to prevent vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD).
Crizanlizumab (SEG101) is a humanized anti-P-selectin monoclonal antibody being studied to restrict VOCs in SCD.
The status has been provided based on positive data from the phase II Sustain study, which compared the P-selectin inhibitor crizanlizumab against placebo in patients with SCD.
The study demonstrated that crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3% compared to placebo in patients with or without hydroxyurea therapy, said the company.
Sustain study also showed that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs against placebo during treatment.
Crizanlizumab is said to bind to a P-selectin molecule on the surface of platelets and endothelium in the blood vessels, and demonstrated to inhibit interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes, enabling to prevent cells from bind to P-selectin.
Novartis Oncology global drug development head Dr Samit Hirawat said: “Painful sickle cell crises matter because they can disrupt patients’ lives, and often require hospital visits and medical attention.
“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.”
Sickle cell disease is a debilitating genetic blood disorder, which alters the shape of red blood cells and can turn blood cells and blood vessels stickier than usual.
Clusters in the bloodstream will be formed when blood cells stick to one another, and these clusters can block the flow of blood and oxygen that may affect blood vessels and organs.
Also called as sickle cell pain crises, VOCs are uncertain and extremely painful events that can create serious acute and chronic complications.
VOCs will result when multiple blood cells stick to each other and blood vessels, causing blockages.