FDA grants breakthrough therapy status to crizanlizumab

Novartis has secured breakthrough therapy designation from the US Food and Drug Administration (FDA) for the use of crizanlizumab (SEG101) to prevent vaso-occlusive crises (VOCs) in sickle cell disease (SCD) patients of all genotypes.

VOCs are unpredictable and extremely painful and can result in serious acute and chronic complications. These events can be treated with therapies that make blood cells and vessels less sticky.

Crizanlizumab is an investigational, humanised anti-P-selectin monoclonal antibody that binds to P-selectin on platelets and endothelium in the blood vessels.

The candidate was found to block interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes, preventing their binding to P-selectin.

"We look forward to working closely with the FDA over the coming months toward making crizanlizumab available in the US as soon as possible."

Novartis Oncology global drug development head 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."

The FDA decision comes after a review of data from the Phase II SUSTAIN clinical trial performed to compare crizanlizumab with placebo in patients suffering from sickle cell disease.

Data revealed a 45.3% decrease in median annual rate of VOCs leading to healthcare visits when treated with crizanlizumab in patients with or without hydroxyurea therapy.

The investigational product also significantly increased the proportion of participants who did not experience any VOCs compared to placebo.

Treatment-emergent adverse events (AEs) and serious AEs were found to be similar to that of placebo. Discontinuations related to adverse events were also low during the trial.

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