FDA News

The FDA accepted fenebrutinib's new drug application under priority review for relapsing and primary progressive MS, based on 3 phase 3 trials comparing the BTK inhibitor with teriflunomide and ocrelizumab.

FDA approves once-daily oral tavapadon for Parkinson disease, a first-in-class D1/D5 partial agonist that improves motor function and ON time, with key safety warnings.

The FDA extended its review of relutrigine for SCN2A- and SCN8A-related epilepsies by three months, pushing the PDUFA date to December 27, 2026.

The FDA has granted Scholar Rock's apitegromab fast track designation for the treatment of patients with living facioscapulohumeral muscular dystrophy as the company's phase 2 FORGE study dosing begins.

Xenon Pharmaceuticals submitted an FDA new drug application for azetukalner, a KV7 potassium channel opener, as adjunctive therapy for focal seizures in epilepsy.

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.

The FDA clearance allows AbelZeta to study bispecific CAR T-cell therapy C-CAR168 in refractory progressive multiple sclerosis.

Microstructure Imaging has announced that the FDA cleared its MICSI-PET, an MR-guided PET enhancement platform with automated amyloid Centiloid and tau quantification.

The FDA granted priority review to satralizumab for MOGAD, positioning it to become the first approved treatment for the rare autoimmune disease.

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

FDA approved Zanvastro (zilganersen), the first therapy to directly target the GFAP protein buildup that drives Alexander disease, based on a phase 3 trial showing improved walking speed and motor function versus untreated controls.

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

Cellenkos’ investigational allogeneic, cord blood-derived T-regulatory cell therapy is being evaluated in a phase 1/1b study in ALS, with early findings showing reductions in plasma neurofilament light chain and increases in IL-10.

The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data.

The FDA has cleared Roche's Elecsys pTau217, a blood test that uses a single biomarker to both rule in and rule out amyloid pathology in adults being evaluated for Alzheimer disease, across both primary and specialty care settings.

FDA has cleared C2N Diagnostics' PrecivityAD2 blood test for adults 40 years and older with cognitive symptoms, offering fast amyloid and tau insight to guide diagnosis and trials in Alzheimer disease.

The FDA granted Fast Track designation to Acadia Pharmaceuticals’ investigational remlifanserin for hallucinations and delusions associated with Alzheimer disease psychosis.

The FDA approved Tauklarify (florquinitau F 18 injection), a tau PET imaging agent, for identifying tau neurofibrillary tangle pathology in adults being evaluated for Alzheimer disease.

The FDA has granted 510(k) clearance to VoxNeuro's Cognitive Function Neuroimaging software, an EEG/ERP-based tool designed to give clinicians an objective, adjunctive measure of cognitive function.

Six FDA PDUFA decisions this fall could reshape care in Alzheimer disease, DMD, Sanfilippo syndrome, Alexander disease, and SMA, here's what clinicians need to know about the data behind each.

The FDA has approved oveporexton, marketed as Orzeyful, as the first therapy to directly restore orexin signaling and address the full symptom range of narcolepsy type 1.

The FDA has accepted Saol Therapeutics' resubmitted NDA for SL1009 in pyruvate dehydrogenase complex deficiency, setting a new PDUFA target action date of December 30, 2026.

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

The nonbinding vote casts doubt on the therapy's path to approval ahead of an August 22 FDA decision, capping over a year of regulatory back-and-forth over the strength of its phase 3 data.

The submission is backed by phase 3 FoCus trial data showing ALXN1840 tripled copper mobilization and produced significant, sustained neurologic improvement compared with standard of care over 48 weeks.

The FDA granted priority review to zeleciment rostudirsen for exon 51-amenable Duchenne muscular dystrophy, with a PDUFA target action date of January 21, 2027.

If approved, AD109 could become the first medication to address both the nighttime airway obstruction and oxygen deprivation central to OSA, as well as its daytime symptoms, such as fatigue.