News from the FDA/CDC

FDA approves treatment for sickle cell pain crises


The Food and Drug Administration has approved crizanlizumab-tmca (Adakveo) to reduce the frequency of vaso-occlusive crisis, a common complication of sickle cell disease.

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The drug is approved for patients aged 16 years and older. It was approved on the strength of the SUSTAIN trial, which randomized 198 patients with sickle cell disease and a history of vaso-occlusive crisis to crizanlizumab or placebo. Patients who received crizanlizumab had a median annual rate of 1.63 health care visits for vaso-occlusive crises, compared with patients who received placebo and had a median annual rate of 2.98 visits. The drug also delayed the first vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months, according to the FDA.

“Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement. “Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease.”

Common adverse events associated with crizanlizumab included back pain, nausea, pyrexia, and arthralgia. The FDA advised physicians to monitor patients for infusion-related reactions.

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