Crizanlizumab is breakthrough therapy for VOCsMarch 7, 2019
The US FDA has granted breakthrough therapy designation crizanlizumab (SEG101) for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD), Novartis said.
The designation was granted based on positive results of 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 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 during treatment.
Also known as sickle cell pain crises, VOCs are unpredictable and extremely painful events that can lead to serious acute and chronic complications.