Posted by Michael Wonder on 08 Jan 2019
Novartis investigational therapy crizanlizumab (SEG101) receives FDA breakthrough therapy designation for the prevention of vaso-occlusive crises in sickle cell disease
8 January 2019 - FDA filing of crizanlizumab anticipated in first half of 2019.
Novartis announced today that the US FDA has granted crizanlizumab (SEG101) breakthrough therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease.
The FDA granted Breakthrough Therapy designation for crizanlizumab based on positive results of the Phase II SUSTAIN trial, which compared the P-selectin inhibitor crizanlizumab with 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 (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) during treatment.
