FDA News

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.

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.

If approved, lecanemab-irmb would provide an at-home subcutaneous injection option for both treatment initiation and maintenance dosing for patients with early-stage Alzheimer disease.

Alixorexton, an oral investigational selective orexin 2 receptor agonist, is currently in development for the treatment of narcolepsy type 1 and type 2, and idiopathic hypersomnia.

The FDA requested the full data from the phase 3 HOPE-3 study as part of its review of the biologics license application for deramiocel, which Capricor plans to submit in February 2026.

Stephanie Fradette, PharmD, head of neuromuscular development at Biogen, gave immediate reaction to the EC approval of high-dose nusinersen and its implications for evolving SMA treatment strategies.

The FDA approved Sentynl Therapeutics’ copper histidinate for the treatment of pediatric Menkes disease, a rare genetic neurodegenerative disorder, following an earlier complete response letter.

The FDA is expected to have a decision on efgartigimod as potentially the first approved therapy for seronegative myasthenia gravis by May 10, 2026.

Catch up on any of the neurology news headlines you may have missed in December 2025, compiled into 1 place by the NeurologyLive® team.

The newly approved generic product is expected to perform like other high-quality glatiramer acetate generics, with differences confined mainly to the manufacturer and device, not mechanism or dosing.

Explore anticipated FDA decisions on groundbreaking neurology drugs in 2026, shaping treatment options for various neurologic conditions.

These were the most impactful FDA approval stories that were part of our coverage in 2025, brought to you as part of NeurologyLive's Year in Review.

The expanded indication brings n-butyl cyanoacrylate’s established performance into the treatment of chronic subdural hematoma, a condition for which traditional surgical interventions may not always be suitable or effective.

Inebilizumab is the first CD19-targeted B-cell therapy approved for antiAChR- or antiMuSK antibody–positive generalized myasthenia gravis, providing symptom control with twice-yearly dosing.

The phase 3 study aims to confirm findings from the HEALEY ALS platform trial in patients with early-stage rapidly progressive ALS, which suggested potential benefits of pridopidine across multiple domains.

Catch up on any of the neurology news headlines you may have missed in November 2025, compiled into 1 place by the NeurologyLive® team.

TRN-257, a treatment in development for adults with narcolepsy and idiopathic hypersomnia, is designed to reduce daily sodium exposure relative to existing higher-sodium therapies.