Rare Diseases: Neuromyelitis Optica, Relapses Blocked Thanks to Ravulizumab

The monoclonal antibody ravulizumab, developed by Alexion – AstraZeneca Rare Disease for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults positive for the anti-aquaporin-4 (AQP4) antibody (Ab+), a rare autoimmune disease that affects approximately 1,500-2,000 people in Italy, is now available and reimbursed, following the green light from AIFA. Safe and effective in reducing relapses in almost all cases, as demonstrated by the phase 3 Champion – NMOSD study, ravulizumab is the first long-acting C5 complement inhibitor for the treatment of this rare disease.