The US Food and Drug Administration (FDA) has approved ZANVASTRO (zilganersen) for the treatment of Alexander disease (AxD) in paediatric and adult patients, providing the first disease-modifying treatment for this ultra-rare and progressive neurological disorder.
Zilganersen is an RNA-targeted medicine designed to reduce production of GFAP, addressing an underlying mechanism of the disease. The 50 mg treatment is administered by intrathecal injection every three months.
The FDA approval was supported by results from a pivotal clinical study. In participants aged five years and older, zilganersen met its primary endpoint, demonstrating statistically significant stabilisation of gait speed compared with control at week 61, measured using the 10-Metre Walk Test. The least-squares mean difference was 33.3%, with a p value of 0.041.
In children aged two to four years, the treatment also improved gross motor function compared with control, assessed using the Gross Motor Function Measure-88. Patient, caregiver and clinician-reported measures also generally favoured zilganersen.
The treatment had a favourable safety and tolerability profile, with most adverse events described as mild or moderate. Serious treatment-emergent adverse events occurred less frequently in the treatment group than in the control group.
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The FDA approval was accompanied by a Rare Pediatric Disease Priority Review Voucher, intended to incentivise the development of treatments for serious rare diseases.
ZANVASTRO is expected to become available in the US in the coming weeks. Ionis has licensed rights outside the US to Recordati, with regulatory submissions in Europe and Japan expected in 2027.
This news item has been summarised using AI and checked by humans before publication.
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