FDA grants breakthrough therapy status for Novartis' crizanlizumab to prevent vaso-occlusive crises in sickle cell disease

Novartis announced Tuesday that the FDA granted crizanlizumab a breakthrough therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD). Samit Hirawat, head of oncology global drug development at the company, said "we look forward to working closely with the FDA over the coming months toward making crizanlizumab…available in the US as soon as possible." 

Novartis indicated that the designation was supported by data from the Phase II SUSTAIN trial. Results of the study, published in the NEJM in 2016, showed that crizanlizumab, also known as SEG101, reduced the median annual rate of VOCs leading to healthcare visits by 45.3 percent compared to placebo in SCD patients with or without hydroxyurea therapy. A post-hoc analysis released last October demonstrated that a significant 35.8 percent of crizanlizumab-treated patients did not experience a VOC during treatment, compared with 16.9 percent for placebo.

Novartis expects to file an FDA application for the experimental drug, a humanised monoclonal antibody targeting P-selectin, in the first half of the year.