New analysis finds Uplizna effective among European populations with neuromyelitis optica spectrum disorder.- Horizon Therapeutics

Uplizna received marketing authorization from the European Commission (EC) on April 25, 2022 and is the first and only targeted CD19+ B-cell-depleting monotherapy proven to reduce attacks in adult patients with NMOSD who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+). This post hoc analysis compared attack rates, disability-related outcomes and safety among 50 trial participants from the European Union (EU) (including participants from Bulgaria, Czech Republic, Estonia, Germany, Hungary and Poland) versus 163 non-EU participants.

Key analysis findings :Participants in the EU who were treated with Uplizna experienced fewer attacks (12.5%) compared to those treated with placebo (30%), sharing similar results with non-EU participants receiving Uplizna (10.7%) or placebo (45.2%). ii. No significant differences in Expanded Disability Status Scale (EDSS) worsening were found between participants in the EU (15%) versus non-EU participants (14.9%). iii. Fewer NMOSD-related hospitalisations were reported among those receiving Uplizna compared to those treated with placebo (mean, EU: 1.0 vs 2.0; non-EU: 1.0 vs 1.33).

“The Uplizna pivotal trial is the largest in NMOSD and clearly demonstrates the merits of targeting CD19 B-cells, including plasmablasts and plasma cells, to provide broad, deep and durable B-cell depletion,” said Karl Boegl, M.D. Ph.D., executive director, EMEA regional medical affairs lead, Horizon. “We believe these data provide treating physicians with greater certainty that a targeted monotherapy like UPLIZNA can be a valuable option for the treatment of NMOSD patients in Europe.”