Healthcare company Novartis revealed on Friday the receipt of approval from the US Food and Drug Administration (FDA) for Adakveo to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and paediatric patients aged 16 years and older with sickle cell disease.

Sickle cell disease is a complex and debilitating genetic blood disorder that goes beyond sickle-shaped red blood cells. The disease is associated with chronic inflammation, causing higher levels of cell adhesion proteins, including P-selectin, which make both the blood vessels and certain blood cells stickier and prone to multicellular interactions, or clusters, in the bloodstream.

The company said Adakveo (crizanlizumab-tmca), previously known as SEG101, is indicated to reduce the frequency of VOCs, or pain crises, in adults and paediatric patients aged 16 years and older with sickle cell disease. It is the first and only targeted biologic that works by binding to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.

Adakveo reportedly represents the first US FDA-approved medicine in sickle cell disease and is expected to be available to patients in the coming weeks, revealed the company.

Based on results of the company's 52-week, randomized, placebo-controlled SUSTAIN trial,Adakveo 5 mg/kg significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo, 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.

About 100,000 people in the US, most of whom are of African descent, have sickle cell disease. The FDA approval comes approximately two months ahead of its priority review action date, allowing Adakveo to be available to patients more quickly, concluded the company.