Ionis wins FDA approval for first Alexander disease drug — SkimNews
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- Ionis Pharmaceuticals received FDA approval Thursday for Zanvastro, the first disease-modifying therapy for Alexander disease.
- The drug was cleared for use in both children and adults following a pivotal trial in which treated patients' walking speed remained stable.
- Patients in the control group experienced a 33% decline in walking speed — a standard motor-function measure in neurologic disease — versus the stable trajectory in those receiving Zanvastro.
- The trial contained hints that treating young children with the drug can improve motor function rather than merely stabilize it.
- Researchers found the drug generally safe, with serious adverse events more common in the control group than among Zanvastro-treated patients.
Why it matters: Patients with Alexander disease — a rare, fatal neurological disorder with no prior approved therapy — now have a disease-modifying option that stabilized walking speed in a pivotal trial while the control arm declined 33%, with early signals that intervention in young children may go beyond stabilization to actual motor-function gains.
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