FDA accepts file and accelerates review of Novartis sickle cell disease medicine crizanlizumab (SEG101)
- FDA grants crizanlizumab Priority Review based on Phase II data showing prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease, shortening FDA review to six months from standard ten months
- Vaso-occlusive crises (also called sickle cell pain crises) are unpredictable and extremely painful events that can lead to serious life-threatening complications and death[1]
- A greater than two-fold increase in the percentage of patients who did not experience any VOCs vs placebo (36% vs 17%,?P=0.010)[3]
- A three-fold longer median time to first VOC vs placebo (4.07 vs 1.38 months,?P< 0.001)
- A 42% reduction in median annual rate of days hospitalized vs placebo (4.00 vs 6.87?P=0.45)
- SOLACE-adults?(A2202) Phase II study investigating the pharmacological properties and safety of crizanlizumab in patients with sickle cell disease aged 16 and above
- SOLACE-kids?(B2201) Phase II study investigating the appropriate dosing, safety, and efficacy of crizanlizumab in pediatric patients with sickle cell disease
- STAND?(A2301) Phase III study investigating the efficacy and safety of crizanlizumab in sickle cell disease patients aged 12 and above
- SUCCESSOR?retrospective cohort study among adult sickle cell disease patients in the US