Sickle Cell Disease Treatment Granted Breakthrough Therapy Designation

Officials with the FDA have granted Breakthrough Therapy designation to Novartis’ crizanlizumab (SEG101) for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD), according to a press release.

Patients with SCD face a high economic burden, with annual costs of more than $30,000 for adults with the disease, according to Novartis. SCD can lead to VOCs, which are painful complications caused by clusters of cells that block or reduce blood flow.

“Painful sickle cell crises matter because they can disrupt patients’ lives, and often require hospital visits and medical attention,” Samit Hirawat, MD, head of Novartis Oncology Global Drug Development, said in a statement. “We look forward to working 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.”

Crizanlizumab, a monthly infusion, is a P-selectin inhibitor that reduces VOCs by binding to a molecule called P-selectin on the surface of platelets and endothelium in the blood vessels. It has been shown to inhibit interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes.

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