Alexander disease

FDA Approves Ionis Pharma Drug, the First for Ultra-Rare Alexander Disease

Ionis Pharmaceuticals’ zilganersen, brand name Zanvastro, is the first disease-modifying therapy approved for the neurological disorder Alexander disease. While Ionis has experience developing medicines for rare neuroscience indications, Zanvastro will be the first neurology product the company brings to the market without a commercialization partner.

By Frank Vinluan

BioPharma, Pharma

Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study

Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners.

By Frank Vinluan