Safety Considerations for Ravulizumab in NMOSD From Open-Label Extension Data: Sean Pittock, MD

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"We also know that different age groups may be predisposed to developing meningococcal infection. That’s something to take into account. With any medication, patients and physicians have to weigh the potential pros—in the case of ravulizumab, it’s a wonderful efficacy—with the potential risks that go along with this medication."

In March, the FDA approved ravulizumab-cwvz (Ultomiris; Alexion), a terminal compliment C5 inhibitor, for the treatment of patients with anti-aquaporin (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). With the decision, the agent became the fourth approved therapy for this patient population, following behind eculizumab (Soliris; Alexion), inebilizumab (Uplizna; Horizon), and satralizumab (Enspryng; Genentech).

The therapy was approved based on data from the phase 3 CHAMPION-NMOSD study (NCT04201262), a large-scale trial that showed no relapses in patients with NMOSD over a 73-week treatment duration. In the trial, the statistically significant and clinically meaningful reductions in the risk of relapse were compared with those on placebo from the external PREVENT rial (NCT01892345), a phase 3 study that evaluated eculizumab. Following the primary treatment period, which lasted up to 2.5 years, patients entered a long-term extension.

At the 2024 American Academy of Neurology (AAN) Annual Meeting, held April 13-18, in Denver, Colorado, new interim data from the open-label extension showed that no patients treated with ravulizumab experienced an adjudicated relapse over a 138.4-week median follow-up. In addition, most patients (91.4%; 53 of 58) had improved HAI score, and most (91.4%; 53 of 58) had no clinically important worsening in Expanded Disability Status Scale score. The analysis, led by Sean Pittock, MD, demonstrated the long-term clinical benefit of ravulizumab in AQP4+ NMOSD.

Pittock, director of the Mayo Clinic Center for Multiple Sclerosis and Autoimmune Neurology, sat down with NeurologyLive® to discuss the safety profile of ravulizumab and specifically, the risk of meningococcal infection. He discussed the importance of following the medication close to its label description and why patients should have appropriate vaccinations before receiving the therapy. Furthermore, Pittock stressed this can be an effective and well-tolerated therapy when administered correctly.

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REFERENCE
1. Pittock S, Barnett M, Bennett J, et al. Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+ NMOSD): interim analysis from the ongoing phase 3 CHAMPION-NMOSD trial. Presented at: 2024 AAN Annual Meeting; April 13-18; ABSTRACT 002489