ULTOMIRIS® (ravulizumab-cwvz) has recently obtained approval in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients diagnosed with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

This approval from the US Food and Drug Administration (FDA) was granted based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology. The trial compared ULTOMIRIS to an external placebo arm from the pivotal SOLIRIS® PREVENT clinical trial.

In the CHAMPION-NMOSD trial, ULTOMIRIS met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Notably, ULTOMIRIS patients experienced zero relapses over a median treatment duration of 73 weeks, resulting in a significant reduction in relapse risk (98.6% reduction, hazard ratio (95% CI): 0.014 (0.000, 0.103), p<0.0001).

Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves. It is characterized by unpredictable relapses, often severe and recurrent, which can lead to permanent disability.

Dr. Sean J. Pittock, Director of Mayo Clinic's Center for Multiple Sclerosis and Autoimmune Neurology and lead primary investigator in the CHAMPION-NMOSD trial, emphasized the significance of C5 inhibition in reducing the risk of NMOSD relapses. He highlighted the approval of ULTOMIRIS as a long-acting C5 inhibitor offering patients a treatment option with demonstrated efficacy in relapse prevention.

Marc Dunoyer, CEO of Alexion, expressed pride in delivering a transformative treatment option for NMOSD patients with ULTOMIRIS. He underscored the company's commitment to advancing science for rare diseases and thanked the NMOSD community for their collaboration.

Safety and tolerability of ULTOMIRIS in the CHAMPION-NMOSD trial were consistent with previous studies, with no new safety concerns identified. Common adverse events included COVID-19, headache, back pain, arthralgia, and urinary tract infection.

ULTOMIRIS is also approved for certain adults with NMOSD in Japan and the European Union (EU), with regulatory reviews ongoing in additional countries.

 

Source: businesswire.com

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