Adakveo FDA approval : Swiss drugmaker Novartis has bagged approval for Adakveo (crizanlizumab-tmca) from the US Food and Drug Administration (FDA) for its use in sickle cell disease.

Formerly known as SEG101, Adakveo has been indicated to be used for bringing down the frequency of vaso-occlusive crises (VOCs), or pain crises, in patients, aged 16 years and older with the genetic blood disorder.

According to the FDA, vaso-occlusive crisis is a usual and painful complication of sickle cell disease caused by blocking of blood circulation by sickled red blood cells. Sickle cell disease is a genetic blood condition in which the red blood cells are shaped abnormally like a sickle. As a result, the sickle-shaped blood cells curb the flow in blood vessels and limit oxygen delivery to the body’s tissues, resulting in severe pain and organ damage.

Adakveo is now the first FDA-approved sickle cell disease drug that binds to P-selectin, a cell adhesion protein that is considered to have a key role in the multicellular interactions that can result in vaso-occlusion.

Brett P. Giroir – Acting FDA Commissioner, commenting on Adakveo FDA approval, said: “Hope has never been higher for people living with sickle cell disease and their families and supporters, with a pipeline of new treatments on the horizon, like the one being approved today, and several initiatives underway to better utilize current tools in the battle against the painful and deadly blood disorder.

“The opportunity before us in the coming months and years is profound and historic.”

Basis of Adakveo FDA approval

Adakveo FDA approval was driven based on the findings of the SUSTAIN clinical trial featuring 198 sickle cell disease patients with a history of vaso-occlusive crisis. The randomized, placebo-controlled trial showed that the Novartis drug delivered a significant reduction in the median annual rate of VOCs to 1.63 vs 2.98 compared to placebo, which represents a reduction in frequency by 45%.

Novartis said that the reductions in the frequency of VOCs were noted among patients irrespective of sickle cell disease genotype and/or hydroxyurea use.

Commenting on Adakveo FDA approval, Susanne Schaffert – Novartis Oncology President, 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.”

Recently, Novartis’ generic drug business Sandoz signed a deal to acquire the Japanese business of Aspen Global Incorporated (AGI) in a deal valued up to €400 million.

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