FDA green light for Zanvastro in Alexander disease

The US Food and Drug Administration (FDA) has approved US biotech Ionis Pharmaceuticals’ (Nasdaq: IONS) Zanvastro (zilganersen) for the treatment of Alexander disease (AxD) in pediatric and adult patients.

Zanvastro is the first and only disease modifying treatment for AxD, an ultra-rare, progressive and often fatal neurological disorder that can affect motor, cognitive, autonomic and gastrointestinal function. Until now, treatment of AxD has primarily been limited to managing symptoms. Zanvastro is an RNA-targeted medicine designed to address the underlying disease mechanism of AxD by reducing the production of glial fibrillary acidic protein (GFAP). Zanvastro 50mg is administered quarterly as an intrathecal (IT) injection.