The U.S. Food and Drug Administration (FDA) approved Adakveo (crizanlizumab-tmca) to reduce the frequency of pain crises in individuals with sickle cell disease. Adakveo (Novartis) is indicated for patients ages 16 or older is a humanized anti-P-selectin monoclonal antibody administered via monthly infusion. It binds to P-selectin on the surface of platelets and endothelium in the blood vessels.

In a press release, Acting FDA Commissioner Adm. Brett P. Giroir, MD 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.”

Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. This rare disease is an inherited blood disorder in which the red blood cells are abnormally shaped (in a crescent or “sickle” shape), which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe chronic inflammation that results in vaso-occlusive crisis where patients experience episodes of extreme pain and organ damage. According to the Centers for Disease Control and Prevetion, sickle cell disease affects approximately 100,000 Americans. The disease occurs most often in African-Americans, where 1 out of every 365 babies born have the disease.

The Adakveo approval was based on the results of a randomized clinical trial enrolling 198 patients with sickle cell disease with a history of vaso-occlusive crisis. Patients either received Adakveo or a placebo. The patients treated with Adakveo experienced fewer health care visits for vaso-occlusive crisis annually (median annual rate of 1.63 visits), compared to patients who received a placebo (median annual rate of 2.98 visits). In addition, 36 percent 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.

Common side effects for patients taking Adakveo were back pain, nausea, pyrexia (fever) and arthralgia (joint pain). Health care professionals are advised to monitor patients for infusion-related reactions and to discontinue Adakveo for severe reactions. Patients who receive Adakveo should be monitored for interference with automated platelet counts or platelet clumping (platelet counts reported may be much lower than the actual count in the blood). Health care professionals are advised to run tests as soon as possible or use citrate tubes (a practice to avoid platelet activation).

The FDA granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.

 

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