ZANVASTRO™ (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients
The FDA approved Ionis Pharmaceuticals' ZANVASTRO (zilganersen) as the first and only disease-modifying treatment for Alexander disease, an ultra-rare and often fatal neurological disorder, in pediatric and adult patients. Approval was based on the pivotal Phase 1-3 study of 54 patients, which met its primary endpoint with a statistically significant 33.3% least-square-mean benefit in gait speed versus control at Week 61 (p=0.041). Ionis also received a Rare Pediatric Disease Priority Review Voucher alongside the approval, and ZANVASTRO will be available in the U.S. in the coming weeks as the company's first independent launch from its neurology pipeline.
Key figures
- Drug Name
- ZANVASTRO (zilganersen)
- Phase Of Trial
- Phase 1-3 (pivotal MAD study, NCT04849741)
- Dosing
- 50 mg quarterly intrathecal injection
- Axd prevalence
- approximately 1 in 1 to 3 million people worldwide
- Us availability
- in the coming weeks
- Study participants
- 54
- Primary endpoint p value
- 0.041
- Ex us regulatory submissions
- Europe and Japan expected in 2027
- Gait speed ls mean difference
- 33.3%
- Priority review voucher awarded
- Yes
AI analysis
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