FDA approves breakthrough treatment for sickle cell disease

The FDA approved Nov. 15 Novartis' drug, Adakveo, the first targeted therapy approved to treat sickle cell disease.

Adakveo is designed 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.

According to the CDC, approximately 100,000 Americans have sickle cell disease, and it occurs most often in African-Americans, where 1 out of every 365 babies born has the disease.

The clinical trial showed patients treated with Adakveo had a median annual rate of 1.63 healthcare visits for vaso-occlusive crisis, compared to an annual rate of 2.98 visits for patients on a placebo. Additionally, 36 percent of patients did not experience vaso-occlusive crisis during the study, and it delayed the time patients first experienced vaso-occlusive crisis after starting treatment from 1.4 months to 4.1 months.

Common side effects of Adakveo include back pain, nausea, fever and joint pain. Physicians are advised to monitor patients being treated with Adakveo for infusion-related reactions and to discontinue for severe reactions. Patients should also be monitored for interference with automated platelet counts or platelet clumping.

Adakveo is designed for patients 16 years and older.

The treatment was given priority review, breakthrough therapy designation and orphan drug designation.

Read the full news release here.

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