FDA accepts file and accelerates review of Novartis sickle cell disease medicine crizanlizumab (SEG101) - Seite 2

The FDA submission is supported by Phase II results from the SUSTAIN study, which showed that crizanlizumab (5 mg/kg) reduced the median annual rate of VOCs leading to health care visits by 45.3% compared with placebo (1.63 vs 2.98, P=0.010) in patients with or without hydroxyurea. Clinically significant reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype or hydroxyurea use.

The study also demonstrated that crizanlizumab (5 mg/kg) showed: 

• A greater than two-fold increase in the percentage of patients who did not experience any VOCs vs placebo (36% vs 17%, P=0.010)[3]
• A three-fold longer median time to first VOC vs placebo (4.07 vs 1.38 months, P< 0.001) 

A 42% reduction in median annual rate of days hospitalized vs placebo (4.00 vs 6.87 P=0.45)

The most frequently reported adverse reactions (>=10%) in patients (N=111) treated with 5 mg/kg crizanlizumab were back pain, nausea, pyrexia, and arthralgia. The majority of adverse reactions were mild to moderate (Grade 1 or 2). Severe (Grade 3) events were observed for arthralgia and pyrexia 0.9% [1 case] each. No patients discontinued treatment due to adverse reactions based on the analysis. In the SUSTAIN trial, there were no apparent increases in reported overall infections (53.0% vs 53.2%) or neutropenia (3.1% vs 6.5%) adverse events with crizanlizumab treatment compared to placebo.

About Sickle Cell Disease
Sickle cell disease is a debilitating inherited genetic blood disorder that affects the shape of the red blood cells and can make blood cells and blood vessels stickier than usual[4],[5]. When blood cells stick to one another, they can form multicellular adhesion clusters in the bloodstream. These clusters can reduce and block the flow of blood and oxygen, which can cause damage to the blood vessels and lead to acute and chronic complications[4],[6]. These blockages also can lead to painful crises called VOCs, which are considered the clinical hallmark of the disease and the main reason why patients seek medical care in hospitals[4]. The average sickle cell patient in the United States is estimated to face nearly $1 million in total lifetime health care costs[7].