FDA approves new treatments for neurologic conditions, Medscape reports

The U.S. Food and Drug Administration approved a slate of new treatments in September 2026 for rare neurologic conditions, genetic disorders, and chronic movement diseases, Medscape reported. The regulatory actions span therapies for Alexander disease, spinal muscular atrophy, Sanfilippo syndrome type A, ataxia-telangiectasia, and Parkinson’s disease, alongside a new liquid formulation for epilepsy.

Zilganersen Targets Alexander Disease Glial Buildup

Zilganersen, developed by Ionis Pharmaceuticals under the brand name Zanvastro, received approval for both pediatric and adult patients with Alexander disease. It marks the first therapy to directly target the glial fibrillary acidic protein buildup associated with the rare and progressive disorder. Clinical trials showed that patients receiving the antisense oligonucleotide via quarterly spinal injections experienced improved walking speed and motor skills compared to untreated participants.

Apitegromab Addresses Muscle Loss in Spinal Muscular Atrophy

Scholar Rock gained approval for apitegromab-mstn, marketed as Isembyld, for patients aged two years or older with spinal muscular atrophy who are already taking an SMN2-targeted therapy. The antibody blocks myostatin signaling to act directly on muscle loss. During a 52-week trial, patients treated with the drug demonstrated more than twice the likelihood of achieving meaningful motor improvement compared to a placebo group, though researchers observed an increased risk of bone fractures.

Rebisufligene Etisparvovec Introduces First Sanfilippo Syndrome Gene Therapy

Ultragenyx Pharmaceutical secured approval for rebisufligene etisparvovec-hopf, known as Fayuvi, as the first treatment for Sanfilippo syndrome type A. The one-time intravenous gene therapy uses an adeno-associated virus serotype 9 delivery system to restore production of the specific enzyme responsible for breaking down heparan sulfate. Open-label study data indicated that pediatric patients who received the infusion maintained or improved their cognitive function.

Levacetylleucine Treats Ataxia in Ataxia-Telangiectasia Patients

IntraBio received approval for levacetylleucine, branded as Aqneursa, as an oral suspension formulation of the modified amino acid N-acetyl-L-leucine. The medication is authorized for adult and pediatric patients weighing at least 15 kilograms who have ataxia-telangiectasia. A placebo-controlled crossover trial involving 73 patients demonstrated significantly better scores on a standardized ataxia scale, with falls and urinary tract infections noted among the most frequent side effects.

Tavapadon Expands Parkinson’s Disease Treatment Options

AbbVie won approval for tavapadon, marketed as Juvmo, for adults with Parkinson’s disease, establishing the first selective dopamine D1/D5 receptor agonist approved for the condition. The medication introduces a new drug mechanism for Parkinson’s disease in over a decade. Late-stage trials showed that the once-daily pill improved motor symptoms compared to placebo, with nausea, dizziness, and headache reported as the primary side effects.

Lamotrigine Liquid Alternative Enters Epilepsy Market

Azurity Pharmaceuticals obtained approval for a new liquid formulation of lamotrigine, called Lamlique, intended as adjunctive therapy for epilepsy patients aged two years or older. The ready-to-use liquid covers partial-onset seizures, primary generalized tonic-clonic seizures, and Lennox-Gastaut syndrome. The manufacturer stated that the formulation provides an alternative to traditional solid oral dosage forms.