FDA Approves First Targeted Treatment for Sickle Cell Crisis

The U.S. Food and Drug Administration approved Adakveo® (crizanlizumab-tmca) to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease (SCD). This is the first targeted therapy for SCD.

The efficacy of crizanlizumab-tmca was evaluated in 198 patients with SCD from the 52-week, randomized, multicenter, placebo-controlled, double-blind SUSTAIN trial. Patients were randomized 1:1:1 to receive crizanlizumab-tmca 5 mg/kg (n=67), crizanlizumab-tmca 2.5 mg/kg (n=66), or placebo (n=65) administered intravenously over 30 minutes at week zero and two and every four weeks thereafter.

Reduced frequency of VOCs

Patients receiving crizanlizumab-tmca 5 mg/kg had a lower median annual rate of VOC leading to a healthcare visit (primary endpoint) compared with those receiving placebo (1.63 vs. 2.98; P=0.010). Reductions in the frequency of VOCs were observed among patients regardless of SCD genotype and/or use of hydroxyurea.

Overall, 36% of patients receiving crizanlizumab-tmca 5 mg/kg did not experience a VOC compared with 17% in the placebo arm. The median time to first VOC from randomization was 4.1 months with crizanlizumab-tmca 5mg/kg versus 1.4 months with placebo.

The most common adverse events associated with crizanlizumab-tmca were nausea, arthralgia, back pain, and pyrexia.

Patients should be monitored for infusion-related reactions and should discontinue treatment with severe reactions. Patients should also be monitored for interference with automated platelet counts or platelet clumping.