Novartis has announced the US Food and Drug Administration (FDA) approval of Adakveo (crizanlizumab), indicated to reduce frequency of pain crises in individuals living with sickle cell disease.
The drug, previously known as SEG101, has been approved to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with the disease.
The approval marks the first available medicine that binds to P-selectin – a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion, and means that the treatment is expected to be available to patients in the coming weeks.
The company announced that the decision is based on results of the 52-week, randomised, 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, which is equivalent to a 45% reduction.
“We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way,” said Kenneth Ataga, director at the center for Sickle Cell Disease and principal investigator of the SUSTAIN trial.
He continued, “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
Sickle cell pain crises are triggered, in part, by multicellular interactions that form clusters of cells, which can block or reduce the blood flow to organs. They can be frequent and sudden, and are associated with an increased risk of life-threatening complications.