Novartis’ crizanlizumab gets FDA breakthrough designation

The FDA has awarded breakthrough designation to crizanlizumab (SEG101), a monthly humanized anti-P-selectin monoclonal antibody drug to treat sickle cell disease that is being developed by Novartis to prevent the painful vaso-occlusive crises, caused by faulty red blood cells that clump and block blood vessels, leading to organ damage.
A phase 2 study has shown that a monthly dose of crizanlizumab significantly reduced the chance of a patient having a VOC in a 12 month period versus placebo. Of those patients who received crizanlizumab, who had been subject to up to ten VOCs in the previous year, nearly 36% of them were VOC free versus 17% of patients on placebo. The median annual rate of VOCs leading to health care visits was reduced by 45% for those patients being treated with crizanlizumab versus placebo, irrespective of whether patients were also being treated with the current therapy for sickle cell, hydroxyurea.