The approval, based on phase 3 IB1001-303 trial data showing consistent SARA scale improvement, makes levacetylleucine the first treatment approved specifically for ataxia-telangiectasia.
Marketed as Fayuvi, the 1-time AAV9 gene therapy showed sustained cerebrospinal fluid heparan sulfate drops and developmental gains among pediatric patients with mucopolysaccharidosis type IIIA in prior studies.
Amy Waldman, MD, Medical Director of the Leukodystrophy Center at Children's Hospital of Philadelphia, discusses zilganersen's mechanism, safety profile, clinical considerations, and what the first-ever approval for Alexander disease means for the broader leukodystrophy field.
Xenon Pharmaceuticals submitted an FDA New Drug Application for azetukalner, a KV7 potassium channel opener, as adjunctive therapy for focal seizures in epilepsy.