Novartis have announced that the U.S. FDA has granted crizanlizumab breakthrough therapy designation for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease.
Breakthrough therapy designation is awarded to treatments for life-threatening conditions that show a significant improvement over current therapies. In this case, crizanlizumab was granted the designation on the back of results from its Phase II SUSTAIN trial which demonstrated the drug reduced the median annual rate of VOCs leading to healthcare visits by just over 45%.
Sickle cell disease is a genetic blood disorder that affects the shape of red blood cells and can make the cells and vessels ‘stickier’ which results in VOCs as well as the increased likelihood of painful complications.
Dr. Samit Hirawat, said: “We look forward to working closely with the FDA over the coming months toward making crizanlizumab, a therapy that has the potential to prevent sickle cell pain crises, available in the US as soon as possible.”