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Zanvastro cleared for Alexander disease

Sponsor
Ionis Pharmaceuticals $IONS
Drug
Zanvastro · zilganersen
Indication
Alexander disease
More on Ionis Pharmaceuticals ↗ Google News

FDA approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in pediatric and adult patients.

What this means

Zanvastro is a genetic therapy designed to reduce the production of a toxic protein that damages brain cells in Alexander disease. The FDA granted its application a full approval based on clinical trial data. This decision provides the first disease-modifying treatment for patients, and the agency "requires healthcare professionals to order and review a genetic test confirming a GFAP mutation prior to treatment initiation.

Read original at drugs.com ↗
Topic Rare Disease