Ionis’ antisense drug stabilizes ultra-rare disease ahead of FDA decision
BioSpace
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.
pharminent
April 22, 2026 (Last updated: April 22, 2026)
BioSpace
The pivotal study of zilganersen in Alexander disease missed a secondary endpoint, but analysts expect the FDA to approve the asset given the unmet need and overall data.