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# Novartis Gets Breakthrough Status on Sickle Cell Therapy
- URL: https://www.fdaweb.com/novartis-gets-breakthrough-status-on-sickle-cell-therapy/
- Published: 2019-01-08T12:00:00.000Z
- Updated: 2026-09-15T00:53:04.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5143108

FDA has granted Novartis a breakthrough therapy designation for crizanlizumab (SEG101) for use in preventing vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease. The company says VOCs are unpredictable and extremely painful events that can lead to serious acute and chronic complications. They happen when multiple blood cells stick to each other and to blood vessels, causing blockages.

The designation was based on positive results from the Phase 2 SUSTAIN trial, which compared the P-selectin inhibitor crizanlizumab against a placebo in patients with sickle cell disease. SUSTAIN showed that crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3% compared to placebo in patients with or without hydroxyurea therapy, Novartis says. The study also demonstrated that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo during treatment, it says.

Novartis describes crizanlizumab as a humanized anti-P-selectin monoclonal antibody that binds to the P-selectin a molecule 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.