Clinical EfficacyALXN1840 may displace trientine and zinc in subsets of patients and eventually compete for first-line use in newly diagnosed cases due to its superior copper mobilization and convenient once-daily oral dosing.
Market PositionThe data position ALXN1840 as a potential step-change therapy for Wilson disease, offering a significant therapeutic advance for the first time in over three decades.
Safety And TolerabilitySafety and tolerability continue to look clean: In total, the safety analysis of 266 patients demonstrated 13 (4.9%) any drug-related serious adverse events.