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Novartis Receives FDA Breakthrough Therapy Designation for Crizanlizumab

January 10, 2019
In the prevention of vaso-occlusive crises in sickle cell disease patients, the inhibitor led to fewer rates of healthcare visits

A PPM Brief

Novartis has announced1 FDA Breakthrough Therapy designation for crizanlizumab for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD). As per FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life-threatening disease or condition while demonstrating a substantial improvement over existing therapies.

Also known as sickle cell pain crises, VOCs can be unpredictable and extremely painful events that lead to serious acute and chronic complications. These crises happen when multiple blood cells stick together and to blood vessels, causing blockages. Treatments such as crizanlizumab that make blood cells and blood vessels less sticky may help reduce the number of days patients experience VOCs.

In the prevention of vaso-occlusive crises in sickle cell disease patients, the inhibitor led to fewer rates of healthcare visits. (Source: 123RF)

Based on the positive results of a Phase II SUSTAIN trial, researchers compared the P-selectin inhibitor crizanlizumab with placebo in patients with SCD. The trial showed that crizanlizumab reduced the median annual rate of VOCs leading to healthcare visits by 45.3% compared to placebo (1.63 vs 2.98, P = 0.010) in patients with or without hydroxyurea therapy. The study also demonstrated that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo (35.8% vs 16.9%, P = 0.010). In addition, patients taking crizanlizumab (5 mg/kg) experienced a similar incidence of treatment-emergent adverse events (86.4% vs 88.7%) and serious adverse events (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 twice as high as the placebo group included arthralgia, diarrhea, pruritus, vomiting, and chest pain, with no apparent increases in infections.

Last updated on: January 25, 2019
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