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Ionis wins FDA clearance for Alexander disease drug, company's first solo neurology approval

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Ionis Pharmaceuticals wins FDA approval for first Alexander disease treatment, priced at $285,000 per dose

The FDA approved Zanvastro, an antisense oligonucleotide therapy from Ionis Pharmaceuticals, as the first targeted treatment for Alexander disease, a rare and deadly genetic neurological disorder affecting adults and children. The company priced the injectable drug at $285,000 per dose.

Sep 3, 2026·5 sourcesFDA drug approvalDrug pricing
Ionis Pharmaceuticals wins FDA approval for first Alexander disease treatment, priced at $285,000 per dose
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Other sources for this story

Statnews

Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder

Biospace

FDA approves Ionis’ antisense drug as first targeted therapy for Alexander disease

Reuters

Ionis Pharma prices first FDA-approved treatment for Alexander disease at $285,000 per dose | Reuters

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Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline