FDA News

New exon‑51 skipping therapy boosts dystrophin and patient strength, with upcoming trial readouts aiming to support accelerated FDA approval.

The FDA's acceptance and priority review of levacetylleucine for ataxia-telangiectasia positions the therapy to potentially become the first approved treatment for the rare neurodegenerative disorder.

FDA Priority Review of asundexian marks a key regulatory milestone for factor XIa inhibition in secondary stroke prevention following positive phase 3 OCEANIC-STROKE results.

Catch up on any of the neurology headlines you may have missed from last week, compiled into 1 place by the NeurologyLive® team.

The FDA has expanded the use of efgartigimod alfa-fcab and Hytrulo to include adults with AChR-Ab seronegative generalized myasthenia gravis, marking the first approved FcRn-targeted therapy for this patient population.

The FDA approval of ocrelizumab for pediatric RRMS expands access to a high-efficacy anti-CD20 therapy for children and adolescents with historically limited treatment options.

Clene plans to pursue accelerated FDA approval for CNM-Au8 in ALS based primarily on neurofilament light biomarker data and phase 2 studies, with a confirmatory phase 3 trial expected to follow.

Catch up on any of the neurology headlines you may have missed in April 2026, compiled into 1 place by the NeurologyLive® team.

FDA clears AXS-05 to ease Alzheimer dementia agitation, offering an oral option that significantly delays symptom relapse in long-term data.

The FDA has cleared an updated Cala kIQ Plus wearable system, adding adaptive features to support personalized management of hand tremor in essential tremor and Parkinson disease.

Neurology News Network for the week ending April 11, 2026. [WATCH TIME: 4 minutes]

FDA accepts Ultragenyx’s UX111 gene therapy BLA for Sanfilippo A, setting a September 2026 decision date.

Stay informed on the latest therapeutic advancements with this clinical recap from the NeurologyLive® team, featuring a centralized look at 7 FDA-approved treatments from the first quarter of 2026.

Catch up on any of the neurology headlines you may have missed in March 2026, compiled into 1 place by the NeurologyLive® team.

Rachele Berria, MD, PhD, Medical Affairs at Chiesi Global Rare Diseases, discussed the clinical data and implications of newly approved every-4-week dosing of pegunigalsidase alfa for Fabry disease in Europe.

BridgeBio Pharma has submitted a new drug application to the FDA for BBP-418 for the treatment of limb-girdle muscular dystrophy type 2I/R9, supported by interim phase 3 trial data.

The FDA has approved a higher-dose regimen of nusinersen for spinal muscular atrophy, supported by DEVOTE data showing improved motor outcomes, reduced neurodegeneration markers, and a safety profile consistent with prior dosing.

The FDA granted accelerated approval to tividenofusp alfa for Hunter syndrome, introducing the first enzyme replacement therapy designed to cross the blood-brain barrier and target neurologic disease.

New data from the pivotal study of investigational therapy zilganersen in Alexander disease will be presented at the 2026 American Academy of Neurology (AAN) annual meeting in Chicago.

Levacetylleucine met its primary end point in a phase 3 trial of patients living with ataxia-telangiectasia, with no drug-related serious adverse events reported, supporting the sNDA.

The FDA has approved leucovorin calcium (Wellcovorin) as the first treatment for patients with cerebral folate transport deficiency caused by variants in the FOLR1 gene.

Catch up on any of the neurology headlines you may have missed in February 2026, compiled into 1 place by the NeurologyLive® team.

PTC Therapeutics pulls ataluren’s US NDA for nonsense-mutation Duchenne after FDA doubts efficacy, despite trial and registry signals of benefit.

52-week trial data shows inebilizumab sustains gains in generalized myasthenia gravis, cuts prednisone, and spotlights CD

The FDA expands pitolisant, marketed as Wakix, to treat cataplexy in pediatric patients 6 years and older with narcolepsy, making pitolisant the only nonscheduled option for excessive daytime sleepiness and cataplexy attacks.

Catch up on any of the neurology headlines you may have missed in January 2026, compiled into 1 place by the NeurologyLive® team.

The FDA has granted priority review to Takeda’s new drug application for oveporexton, an orexin receptor 2-selective agonist, for narcolepsy type 1, with a PDUFA date in the third quarter of 2026.

The FDA issued a CRL for gene therapy RGX-121 in Hunter syndrome, citing trial design and biomarker doubts; Regenxbio seeks a meeting with the agency and resubmission.