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FDA Approves First Targeted Therapy to Treat Complication of Sickle Cell Disease

November 26, 2019

The FDA granted Novartis approval for Adakveo (crizanlizumab-tmca) for reducing the frequency of vaso-occlusive crisis, a common complication of sickle cell disease that occurs when blood circulation is obstructed by sickled red blood cells, for patients 16 and older.

The approval was based on the results of a randomized clinical trial of 198 patients with sickle cell disease and a history of vaso-occlusive crisis. Patients treated with the drug experienced fewer health care visits for vaso-occlusive crisis annually compared to patients who received a placebo.

In addition, 36 percent of patients on Adaveko did not experience vaso-occlusive crisis during the study, and the treatment delayed the time that patients first experienced vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months.

Adakveo was previously granted priority review, breakthrough therapy and orphan drug designations by the agency.

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