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Alexander disease

Features & Analysis

Ionis priced ZANVASTRO at $285,000 a dose. Can the first Alexander disease drug build a viable ultra-rare franchise?

FDA approval has transformed zilganersen from an experimental antisense programme into the first disease-modifying medicine for Alexander disease. For Ionis Pharmaceuticals, however, ZANVASTRO is also a test of whether years of RNA-targeted neurology research can translate into a sustainable independently commercialized franchise.

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Pharma & Biotech

Ionis wins first FDA approval for Alexander disease. Can Zanvastro make an ultra-rare launch work?

Zanvastro finally gives Alexander disease a targeted therapy. Its next challenge is finding patients and funding a $1.14 million annual regimen.

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