The Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz; Alexion) injection for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)

“Prior to this approval, the only approved therapy for PNH required treatment every 2 weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every 8 weeks, without compromising efficacy,” stated Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

Ultomiris, a long-acting complement inhibitor, prevents hemolysis by specifically binding to the complement protein C5, inhibiting its cleavage to C5a and C5b, and thus preventing the generation of the terminal complement complex C5b9. 

The safety and efficacy of Ultomiris in patients with PNH was assessed in 2 open-label, randomized, active-controlled, non-inferiority Phase 3 studies. In PNH Study 301, patients naïve to complement inhibitor treatment (N=246) were randomized to receive Ultomiris or eculizumab (Soliris; current standard of care). Results showed Ultomiris was noninferior to eculizumab as evidenced by a similar incidence of hemolysis and transfusion avoidance. 

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In PNH Study 302 (N=195), patients with PNH who were clinically stable after having been treated with eculizumab for at least 6 months were randomized to either Ultomiris or to continue eculizumab. The data showed similar results between the Ultomiris and eculizumab groups on clinical measures including hemolysis and transfusion avoidance. 

Headache and upper respiratory tract infection were common adverse reactions associated with Ultomiris. A Boxed Warning in the prescribing information describes the risk of life-threatening meningococcal infections and sepsis. Patients should be immunized with meningococcal vaccines at least 2 weeks prior to receiving the first dose of Ultomiris, unless the risks of delaying treatment outweigh the risks of developing an infection. 

Ultomiris will be available as a 300mg/30mL (10mg/mL) strength preservative-free solution in single-dose vials. 

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