FDA approves Ionis’ Zanvastro as first disease-modifying therapy for Alexander disease

The U.S. Food and Drug Administration has granted approval to Ionis Pharmaceuticals' antisense oligonucleotide Zanvastro, the first therapy that modifies disease progression for Alexander disease.

Zanvastro works by reducing the excess GFAP protein that builds up in brain glial cells, addressing the root cause of the disorder rather than just providing supportive care.

Alexander disease is an ultra-rare leukodystrophy, estimated to affect about one in three million people worldwide, and until now patients had no approved treatment options.

Ionis intends to leverage this breakthrough to seek further approvals for other rare neurodegenerative conditions, including Angelman syndrome.