FDA approves Ionis' ZANVASTRO (zilganersen) for Alexander disease

IONIS PHARMACEUTICALS INC

• ZANVASTRO is first and only disease-modifying treatment for AxD; 50 mg intrathecal quarterly.
• Pivotal study met primary endpoint: gait speed stabilization at Week 61 (LS mean diff 33.3%, p=0.041).
• FDA granted Rare Pediatric Disease Priority Review Voucher with approval.
• Recordati holds ex-U.S. rights; EU/Japan regulatory submissions expected in 2027.
• ZANVASTRO available in U.S. in coming weeks; Ionis' first independent neurology launch.