Adakveo approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease.- Novartis

Novartis announced that the FDA approved Adakveo (crizanlizumab), previously known as SEG 101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease. Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin �a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion. The medicine is expected to be available to patients in the coming weeks.

The FDA�s decision to approve Adakveo 5 mg/kg is based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo (P=.010), which is equivalent to a 45% reduction. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea use.Additional results from the SUSTAIN study include: A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction). Thirty-six percent of patients treated with Adakveo did not experience a VOC, compared to 17% of placebo-treated patients. The median time to first VOC was 4.1 for Adakveo vs 1.4 months for placebo. The most common adverse reactions (incidence > 10%) were nausea (18%), arthralgia (18%), back pain (15%) and pyrexia (11%).