FDA Approves Crizanlizumab for Sickle Cell Complications

The US Food and Drug Administration (FDA) has approved crizanlizumab (Adakveo) as the first therapy indicated for the treatment of painful complications due to sickle cell disease in patients aged 16 years and older.

The approval for the Novartis therapy indicate its use in reducing vaso-occlusive crisis frequency. The condition is a common and painful complication of sickle cell disease, driven by obstructed blood circulation.

The FDA’s approval was based on the results of the 52-week, randomized, placebo-controlled SUSTAIN trial showing crizanlizumab 5 mg/kg significantly lowered the median annual rate of crises to 1.63, compared to the median 2.98 reported by placebo patients (P = .01). Reductions in crises frequency were observed among patients regardless of sickle cell disease genotype or patient hydroxurea use.

SUSTAIN findings also showed the therapy was associated with a 42% reduction in median annual rate of days hospitalized (4 vs 6.87), a 19 percentage-point improvement in rate of patients to not experience a crisis (36% vs 17%), and a 2.7-month increase in median time to first crisis (4.1 vs 1.4).

Common adverse events among patients on crizanlizumab included back pain, nausea, pyrexia, and arthralgia. Healthcare professionals are advised to monitor patients for infusion-related reactions, and to discontinue therapy for severe reactions.

Crizanlizumab was previously granted Priority Review, Breakthrough Therapy, and Orphan Drug designations from the FDA.

“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” SUSTAIN principal investigator Kenneth Ataga, MD, director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center at Memphis, said in a statement. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”