Novartis investigational therapy crizanlizumab (SEG101) receives FDA Breakthrough Therapy designation for the prevention of vaso-occlusive crises in sickle cell disease - Seite 2

Patients taking crizanlizumab (5 mg/kg) experienced a similar incidence of treatment-emergent adverse events (AEs) (86.4% vs 88.7%) and serious AEs (25.8% vs 27.4%) compared to placebo, and a low incidence of discontinuations (3%) due to adverse events. Adverse events that occurred in 10% or more of the patients in either active-treatment group (2.5 mg/kg; 5 mg/kg) and at a frequency that was at least twice as high as that in the placebo group included arthralgia, diarrhea, pruritus, vomiting, and chest pain. There were no apparent increases in infections with crizanlizumab treatment⁴.

About Sickle Cell Disease (SCD)
Sickle cell disease is a debilitating genetic blood disorder that affects the shape of your red blood cells and can make blood cells and blood vessels stickier than usual^1,5. When blood cells stick to one another they can form clusters in the bloodstream. These clusters can block the flow of blood and oxygen, which can cause damage to the blood vessels and organs^1,3. These blockages also can lead to painful crises called vaso-occlusive crises, or VOCs. VOCs are painful complications of the disease and the main reason why patients seek medical care in hospitals¹. Treatment of sickle cell disease is also associated with a high economic burden. The average sickle cell disease patient is estimated to face nearly $1 million in total lifetime health care costs with annual costs of more than $30,000 for adults⁶.

About crizanlizumab (SEG101)
Crizanlizumab (SEG101) is a humanized anti-P-selectin monoclonal antibody being investigated for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD). Crizanlizumab binds to a molecule called P-selectin on the surface of platelets and endothelium in the blood vessels and has been shown to inhibit interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes, causing a blockade and thereby preventing these cells from being able to bind to P-selectin. P-selectin is one of the major drivers of the vaso-occlusive process. Our goal is to deepen understanding of the true impact of VOCs on patients' bodies and lives and to explore how crizanlizumab can help to achieve more pain-crisis-free days for patients with SCD⁴.