FDA批准Ionis Pharmaceuticals的Zanvastro(zilganersen)治疗Alexander病，为首个获批疗法

The US FDA on Thursday approved Ionis Pharmaceuticals' (IONS) Zanvastro (zilganersen) as the first-ever treatment for Alexander disease. The antisense oligonucleotide won approval based on results of a randomized, controlled study of pediatric and adult patients with the condition two years of age and older, and in an open-label substudy of 4 patients below two years old. In patients five years and older, zilganersen improved walking speed compared to those without treatment. In those two to four years old, those who received zilganersen showed improvements in motor skills compared to those in a control group. Alexander disease is a rare neurological disease affecting less than one in a million that is caused by mutations in the gene that produces glial fibrillary acidic protein that progressively damages the nervous system.