Aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+ NMOSD) is a rare autoimmune disease of the central nervous system characterized by recurrent and unpredictable relapses, which can lead to significant and irreversible neurological disability.¹ Ravulizumab is a long-acting C5 complement inhibitor designed to prevent relapses in patients with AQP4+ NMOSD.¹ This interim analysis presents long-term outcomes from the CHAMPION-NMOSD study, evaluating the efficacy and safety of ravulizumab in adults with AQP4+ NMOSD over a median follow-up period of 170.3 weeks.¹ 

Autoantibodies against the acetylcholine receptor (AChR) are often found in patients with generalized myasthenia gravis (gMG), which destroy the post-synaptic membrane of the neuromuscular junction (NMJ) via aberrant complement cascade activation.¹ Humanized monoclonal antibodies with a high affinity to the human terminal complement component C5 such as ravulizumab inhibit the membrane attack complex formation.¹ Previously, the 26-week randomized controlled period (RCP) of the phase 3 CHAMPION MG study demonstrated the efficacy and safety profile of ravulizumab in adult patients with anti-acetylcholine receptor antibody-positive (AChRAb+) gMG.¹

Previously, a univariate retrospective analysis from the International Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry showed that PNH patients with high disease activity (HDA) symptoms and a history of thromboembolism events (TEs) or major adverse vascular events (MAVEs) have a significantly greater risk of developing TEs than those without HDA or a history of events.¹ In fact, TEs are the most common causes of death in PNH patients which account for 40% to 67% of the PNH mortality.²