Shots:
- The US FDA has accepted NDA & granted priority review to zilganersen for the treatment of Alexander disease (AxD), with PDUFA target action date of Sep 22, 2026
- NDA was backed by the P-III trial assessing zilganersen (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for 60wks., followed by an open label period & 120wk. open-label LTE, where 25mg cohort moved to the 50mg, & finally a 28wk. post-treatment follow-up
- Trial showed zilganersen (50mg) met the 1EP of gait speed as assessed by 10-Meter Walk Test at 61wks., plus showed consistent benefit in key secondary & exploratory EPs, incl. adaptive function, communication, GI symptoms, sleep & seizures; additional data to be presented at AAN’26
Ref: Ionis | Image: Ionis | Press Release
Related News: The EC Approves Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)
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