US FDA approves Ionis Pharma's therapy to treat rare brain disorder
IONS•FDA approves Ionis therapy for rare brain disorder
The U.S. FDA on Thursday approved Ionis Pharmaceuticals' IONS.O therapy to treat a rare disease that affects the brain's white matter, making it the first treatment to get the nod for the genetic disorder.
Ionis' drug zilganersen, branded as Zanvastro, can be used to treat adults and pediatric patients with Alexander disease, a neurological disorder caused by mutations in a gene that can cause seizures and delay physical and intellectual development.
The condition affects fewer than 1,000 people in the U.S., according to the National Institutes of Health.
In an early-to-late-stage study, patients who got a 50 mg dose of zilganersen showed a statistically significant improvement in gait speed as assessed by a 10-meter walk test, an assessment of functional mobility, at 61 weeks.
