Ionis Pharmaceuticals launches Zanvastro following landmark FDA clearance
The FDA approval covers both children and adults living with Alexander disease, providing a treatment option that targets the underlying cause rather than focusing solely on symptoms.
USA—Ionis Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for zilganersen, the first disease-modifying therapy approved for Alexander disease, a rare and progressive neurodegenerative disorder.
Ionis will market the antisense oligonucleotide in the United States under the brand name Zanvastro.
The FDA approval covers both children and adults living with Alexander disease, providing a treatment option that targets the underlying cause rather than focusing solely on symptoms.
Targeting the cause of Alexander disease
Alexander disease is a genetic disorder linked to abnormal expression of the glial fibrillary acidic protein (GFAP) gene.
The resulting buildup of abnormal GFAP proteins forms toxic aggregates in astrocytes, cells that support and protect neurons in the brain and spinal cord.
As the disease progresses, patients can develop seizures, muscle weakness, impaired movement, and difficulties swallowing.
Researchers estimate that Alexander disease affects approximately one in one million to three million people worldwide.
Clinical evidence supports approval
The FDA based its decision on results from Ionis’ pivotal study, NCT04849741.
In patients aged five years and older, zilganersen significantly stabilized walking speed, an established measure of motor function, compared with the control group at week 61.
The study also provided supportive evidence in children aged two to four years, suggesting the treatment could benefit a broad age range.
Before Zanvastro, treatment for Alexander disease focused primarily on managing symptoms and complications because no approved therapy addressed the disease’s underlying biology.
The new treatment therefore introduces a different approach for patients and their caregivers.
Amy Waldman, a paediatric neurologist and lead investigator of the pivotal study, said shifting treatment from managing individual symptoms to addressing the biological mechanisms driving Alexander disease could potentially improve outcomes for patients.
Ionis prepares US launch
Ionis plans to make Zanvastro available to patients in the United States in the coming weeks.
The company will support patients and caregivers in navigating insurance coverage and provide information about affordability programmes.
Ionis has not yet publicly disclosed the drug’s price.
Zanvastro will also mark Ionis’ first independently launched neurology medicine.
However, the company will handle commercialisation only in the United States.
Recordati holds ex-US rights
Recordati holds the rights to Zanvastro outside the United States following a licensing agreement with Ionis signed in June 2026.
Under the agreement, Recordati paid Ionis US$30 million upfront and assumed responsibility for regulatory submissions and commercial activities for the medicine in international markets.
The timing of Zanvastro’s launch in markets outside the United States will depend on the regulatory and commercial activities Recordati undertakes.
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