Ionis wins FDA approval for ZANVASTRO
- Ionis Pharmaceuticals (NASDAQ:IONS) received FDA approval for ZANVASTRO (zilganersen) as the first disease-modifying treatment for Alexander disease.
- The treatment showed statistically significant stabilization of gait speed and improvements in motor function measures in clinical studies.
- The FDA granted Ionis a Rare Pediatric Disease Priority Review Voucher alongside the approval.
Ionis Pharmaceuticals (NASDAQ:IONS) announced that the U.S. Food and Drug Administration approved ZANVASTRO (zilganersen) for pediatric and adult patients with Alexander disease, making it the first disease-modifying treatment approved for the condition.
The approval expands treatment options for Alexander disease, an ultra-rare neurological disorder caused by changes in the GFAP gene that lead to abnormal protein accumulation in astrocytes and progressive neurological symptoms.
Ionis Chief Executive Officer Brett P. Monia said, “Today’s approval of ZANVASTRO begins a new chapter for people living with Alexander disease and their families,” highlighting the company’s first independent launch from its neurology pipeline.
The FDA approval was supported by pivotal study results showing ZANVASTRO 50 mg statistically stabilized gait speed in patients aged 5 years and older, with a 33.3% least square mean difference compared with control at Week 61, while younger patients showed improvements in gross motor function measures.
Ionis stated that ZANVASTRO will be available in the U.S. in the coming weeks, with patient support services provided through Ionis Every Step, including education, insurance assistance, and affordability resources.
Ionis develops RNA-targeted medicines for neurological and other serious diseases, and has partnered with Recordati for rights to develop and commercialize zilganersen outside the U.S., with regulatory submissions in Europe and Japan expected in 2027.
