New Sickle Cell Disease Drug Approved by FDA

MONDAY, Nov. 18, 2019 (HealthDay News) -- A new drug to reduce a serious complication of sickle cell disease has been approved by the U.S. Food and Drug Administration.

Adakveo (crizanlizumab-tmca) was approved for use in patients aged 16 and older to reduce the frequency of vaso-occlusive crisis, a common and painful complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells.

The approval of the new Novartis drug is based on the results of a clinical trial that included 198 patients with a history of vaso-occlusive crisis. They received either Adakveo or a placebo. The patients who received Adakveo had fewer health care visits for vaso-occlusive crisis (median of 1.63 visits a year) than patients who received the placebo (median of 2.98 visits a year).

Also, 36% of patients who received Adakveo did not experience vaso-occlusive crisis during the study, and it delayed the time that patients first experienced vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months, according to the FDA.

Common side effects for patients taking Adakveo were back pain, nausea, fever and joint pain.

Sickle cell disease is an inherited blood disorder in which the red blood cells have an abnormal crescent or sickle shape, which restricts the flow in blood vessels and limits oxygen delivery to the body's tissues, resulting in severe pain and organ damage.

Sickle cell disease affects about 100,000 Americans, and occurs most often in black Americans, where 1 out of every 365 babies born have the disease, according to the U.S. Centers for Disease Control and Prevention.

"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," said Dr. Richard Pazdur, director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research.

"Vaso-occlusive crisis can be extremely painful and is a frequent reason for emergency department visits and hospitalization for patients with sickle cell disease," Pazdur said in a FDA news release.

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