Adakveo can be provided as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate, said Novartis.
Adakveo attaches to P-selectin, a cell adhesion protein that plays a crucial role in the multicellular interactions that may cause vaso-occlusion.
The approval is based on data from the SUSTAIN trial, which demonstrated that Adakveo significantly minimised the median annual rate of VOCs to 1.63 against 2.98 compared with placebo. It is equivalent to a 45% reduction.
Adakveo, earlier known as SEG101, is claimed to be the first and only targeted biologic that works by binding to P-selectin.
The drug restricts interactions between endothelial cells, platelets, red blood cells, and leukocytes by binding to P-selectin on the surface of the activated endothelium and platelets.
At present, Adakveo secured approval in 36 countries, including the US and European Union member states.
Novartis Oncology European region head Kees Roks said: “Data shows that nine out of ten people living with sickle cell disease experience one or more VOCs in a year, with a third of those crises leading to hospitalization, underscoring the significant unmet need among a vulnerable group of patients.
“Just one VOC could be catastrophic for the patient, so preventing these sudden, unpredictable and life-threatening events is hugely important. Today’s decision gives people living with sickle cell disease a chance to achieve that goal.”
In August this year, Novartis secured EC approval for its Cosentyx (secukinumab) to treat moderate-to-severe plaque psoriasis in children and adolescents aged between six and 18 years.