The U.S. Food and Drug Administration have accepted the Novartis's biologics license application (BLA) and has granted priority review for its investigational sickle cell medicine crizanlizumab. If FDA-approved, crizanlizumab is expected to represent the first monoclonal antibody targeting the P-selectin mediated multi-cellular adhesion in sickle cell disease.
Novartis submitted the application for crizanlizumab for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD) and was granted breakthrough therapy designation last December. According to Novartis, VOCs are unpredictable and extremely painful events that can lead to serious acute and chronic life-threatening complications and death. VOCs also lead to significant health care utilization. They are the most common cause of emergency room visits and hospital admissions for SCD patients, with total medical costs exceeding $1.1 billion annually in the United States.
"The FDA's decision to give crizanlizumab priority review reflects the impact that this medicine could have for the many thousands of US sickle cell adult patients who experience painful vaso-occlusive crises," said John Tsai, MD, head of global drug development and chief medical officer of Novartis.
The FDA submission is supported by Phase II results from the SUSTAIN study, which showed that crizanlizumab (5 mg/kg) reduced the median annual rate of VOCs leading to health care visits by 45.3% compared with placebo (1.63 vs 2.98, P=0.010) in patients with or without hydroxyurea. Clinically significant reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype or hydroxyurea use.
Read the full Novartis release