FDA Neurology Digest: September 2026

This digest highlights FDA actions in September related to therapeutics for neurologic illness. For information on approvals in other specialties during 2026, see our FDA Drug Approvals page.Alexander Disease Drug ApprovedZilganersen (Zanvastro, Ionis Pharmaceuticals, Inc.) received approval for Alexander disease in pediatric and adult patients — the first therapy to directly target the glial fibrillary acidic protein buildup associated with the rare, progressive disorder. Patients who received the antisense oligonucleotide, delivered by quarterly spinal injection, had improved walking speed and motor skills compared with those who received no treatment in clinical trials.Spinal Muscular Atrophy (SMA) Add-On Targets Muscle LossThe FDA approved apitegromab-mstn (Isembyld, Scholar Rock) for SMA in patients aged 2 years or older already taking an SMN2-targeted therapy. The antibody is the first SMA treatment to act directly on muscle loss by blocking myostatin signaling. In a 52-week trial, patients on the drug were more than twice as likely as those on placebo to show meaningful motor improvement, though researchers noted an increased fracture risk.First Gene Therapy for Sanfilippo ASanfilippo syndrome type A has its first approved treatment: rebisufligene etisparvovec-hopf (Fayuvi, Ultragenyx Pharmaceutical Inc.), a one-time intravenous gene therapy for pediatric patients. The adeno-associated virus serotype 9-based infusion restores production of the enzyme that breaks down heparan sulfate. In an open-label study, children who received treatment maintained or improved cognitive function.New Therapy for Ataxia-TelangiectasiaThe agency approved levacetylleucine (Aqneursa, IntraBio), an oral suspension formulation of a modified amino acid (N-acetyl-L-leucine), to treat ataxia in adult and pediatric patients weighing at least 15 kg (33 lb) with ataxia-telangiectasia. In a placebo-controlled crossover trial of 73 patients, the drug produced significantly better scores on a standardized ataxia scale, with falls and urinary tract infections among the most common side effects.Novel Parkinson’s Disease (PD) Drug ApprovedTavapadon (Juvmo, AbbVie) gained approval for PD in adults, becoming the first selective dopamine D1/D5 receptor agonist approved for the condition and the first new drug mechanism for PD in more than a decade. The once-daily pill improved motor symptoms compared with placebo in late-stage trials, with nausea, dizziness, and headache as the most common side effects.Epilepsy Drug Now in Liquid FormA new liquid formulation of lamotrigine (Lamlique, Azurity Pharmaceuticals) received approval as adjunctive therapy in patients with epilepsy and partial-onset seizures, primary generalized tonic-clonic seizures, and Lennox-Gastaut syndrome in patients aged 2 years or older. The ready-to-use formulation offers an alternative to solid oral dosage forms, the manufacturer said in a statement.This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.