Novartis has secured approval for Adakveo (crizanlizumab) from the US Food and Drug Administration (FDA) for its use in sickle cell disease patients.
Formerly known as SEG101, Adakveo has been approved for reducing the incidents of vaso-occlusive crises (VOCs), or pain crises, in adult patients with sickle cell disease, aged 16 years and older.
The Novartis drug is now the first FDA-approved treatment in sickle cell disease that binds to P-selectin, a cell adhesion protein that plays a key role in the multicellular interactions that possibly lead to vaso-occlusion.
Through its binding to P-selectin on the surface of the activated endothelium and platelets, the humanized anti-P-selectin monoclonal antibody is said to prevent interactions between endothelial cells, platelets, red blood cells, and leukocytes.
Novartis oncology president Susanne Schaffert said: “The approval of Adakveo marks a new era in the treatment of sickle cell disease, a genetic condition that places an extraordinary burden of unpredictable pain crises on patients and their families.
“The stories we have heard from patients about their sickle cell pain crises are devastating. We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”
The drug’s FDA approval was based on the results of the 52-week, randomised, placebo-controlled SUSTAIN trial. During the phase 2 trial, Adakveo was shown to have considerably reduced the median annual rate of VOCs to 1.63 compared to 2.98 recorded in the placebo arm.
According to Novartis, the reductions in the frequency of VOCs were seen in patients regardless of the genotype of their sickle cell disease and/or hydroxyurea use.
University of Tennessee Health Science Center at Memphis center for sickle cell disease director and SUSTAIN trial principal investigator Kenneth Ataga said: “We know this drug can decrease the frequency of sickle cell pain crises in a significant and clinically meaningful way.
“The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”
Adakveo’s approval by the FDA follows the priority review status it received from the regulator in July 2019. Prior to that, the anti-P-selectin monoclonal antibody was granted breakthrough therapy designation from the FDA in January 2019 for the prevention of VOCs in patients of all genotypes with sickle cell disease.