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FDA Approves Adakveo to Reduce Vaso-Occlusive Crises in Patients with Sickle-Cell Disease

Web Exclusives - FDA Updates, In the News

On November 15, 2019, the FDA approved crizanlizumab (Adakveo; Novartis) to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged ≥16 years with sickle-cell disease.

Sickle-cell disease affects approximately 100,000 individuals in the United States, most often African Americans. Individuals with the disease have periodic episodes in which red blood cells adhere to the inner lining of blood vessels, preventing oxygen from reaching small blood vessels and tissues. This causes intense pain (or VOC), as well as organ damage and, in some cases, life-threatening complications. These symptoms are the primary reason for emergency department visits and hospital admissions for patients with sickle-cell disease.

The FDA approval of crizanlizumab was based on data from the randomized, placebo-controlled, double-blind SUSTAIN trial of 198 patients with sickle-cell disease who had 2 to 10 VOC episodes in the 12 months before study enrollment. The primary efficacy end point in the trial was the annual rate of VOCs leading to a healthcare visit.

Patients were randomized to receive crizanlizumab 5 mg/kg (N = 67), crizanlizumab 2.5 mg/kg (N = 66), or placebo (N = 65) administered intravenously on week 0, week 2, and every 4 weeks thereafter. The total treatment duration was 52 weeks.

The results showed that treatment with crizanlizumab significantly lowered the median annual rate of VOCs compared with placebo (1.63 vs 2.98, respectively; P = .01). The median time to first VOC from randomization was 4.1 months with crizanlizumab and 1.4 months with placebo.

The most common (>10%) adverse reactions in patients receiving crizanlizumab were nausea, arthralgia, back pain, and pyrexia.

“Patients with sickle cell disease often face unique challenges, and have long suffered silently through unimaginable pain crises,” said Beverley Francis-Gibson, President and CEO of the Sickle Cell Disease Association of America in a Novartis press release. “We are excited to have a new medicine that may help many of the thousands of people living with sickle cell disease by reducing the frequency of these potentially dangerous and painful episodes.”

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Last modified: April 27, 2020