The Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz; Alexion) for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric patients aged 1 month and older.

The approval was supported by data from 2 global, single-arm open-label studies that evaluated the efficacy of ravulizumab-cwvz in complement inhibitor-naïve patients with aHUS. The ongoing pediatric study included 14 children in the interim analysis, and the adult study assessed a total of 56 patients. The primary end point for both studies was based on Complete TMA Response during an initial 26-week evaluation period, defined by hematologic normalization parameters (platelet count and LDH) and improved kidney function (as measured by ≥25% improvement in serum creatinine from baseline).

Findings from each study demonstrated a Complete TMA Response in 71% (interim data; 95% CI: 0.42, 0.92) of children and 54% (95% CI: 0.40, 0.67) of adults during the initial 26-week treatment period. Additionally, ravulizumab-cwvz treatment resulted in reduced thrombocytopenia in 93% (95% CI: 0.66, 0.99) of children and 84% (95% CI: 0.72, 0.92) of adults; reduced hemolysis in 86% (95% CI: 0.57, 0.98) of children and 77% (95% CI: 0.64, 0.87) of adults; and improved kidney function in 79% (95% CI: 0.49, 0.95) of children and 59% (95% CI: 0.45, 0.72) of adults.

With regards to safety, the most common treatment-emergent adverse events were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension, and pyrexia. Ultomiris carries a Boxed Warning for severe meningococcal infections and is only available under a Risk Evaluation and Mitigation Strategy (REMS) program that prescribers are required to enroll in.

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“The primary approach to treatment is to prevent the body from attacking itself, through the inhibition of uncontrolled complement activation, referred to as C5 inhibition,” said Spero Cataland, MD, Professor of Clinical Internal Medicine, Wexner Medical Center, The Ohio State University College of Medicine. “Clinical study results showed adult and pediatric patients had complete C5 inhibition following the first dose of Ultomiris. C5 inhibition was sustained over time with only six or seven infusions a year in adults – and that is important to consider for my patients.”

Ultomiris is already FDA-approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults. The product is available as a 300mg/30mL (10mg/mL) strength preservative-free solution in single-dose vials. 

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