FDA approves first therapy to reduce vaso-occlusive crisis in sickle cell disease

FDA has approved first therapy to reduce the frequency of vaso-occlusive crisis, a painful complication of sickle cell disease in patients above 16 years of age.

The U.S. Food and Drug Administration has approved crizanlizumab (Adakveo) as the first therapy indicated for the treatment of painful complications due to sickle cell disease in patients aged 16 years and older. The treatment reduces 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 FDA has granted approval of Adakveo to Novartis.

According to the Centers for Disease Control and Prevention, 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. Sickle cell disease is an inherited blood disorder in which the red blood cells are abnormally shaped. These crescent or "sickle" shape cells restrict the flow in blood vessels limiting oxygen delivery to the body’s tissues leading to severe pain and organ damage.

“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,” said Acting FDA Commissioner Adm. Brett P. Giroir, M.D. “The opportunity before us in the coming months and years is profound and historic.”

“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 Richard Pazdur, M.D., 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.”

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 per cent of patients who received Adakveo did not experience a 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. Adakveo also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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